2,248 Matching Annotations
  1. Last 7 days
    1. PatientID: None

      KindredID: 67

      Case: Sex Unknown, Age Unknown, Japanese

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0009711; HP:0010797; HP:0005584; HP:0001732 (Retinal capillary hemangioma, Hemangioblastoma, Renal cell carcinoma, Abnormality of the pancreas)

      CaseHPOFreeText: CNS hemangioblastoma, retinal angioma, pancreatic cyst or tumor (unspecified) and renal cell carcinoma.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.L198Gfs*57 (c.592_593del)

      LegacyVariant: c.805_806del

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA658795185, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.592_593del

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: deletion; minus_2_framshift_variant

      CivicName: L198Gfs*57 (c.592_593del)

      MultipleGeneVariants: N/A

    2. GroupID: 9

      DiseaseAssertion: VHL

      GroupSize: 3

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: Japanese

      GroupAgeRange: N/A

      GroupInfo: 3 individuals from a single family (ID 9).

      GroupPresentingHPOs: HP:0009711; HP:0010797; HP:0001732 (Retinal capillary hemangioma, Hemangioblastoma, Abnormality of the pancreas)

      GroupHPOFreeText: CNS hemangioblastoma (3/3), retinal angioma (3/3) and pancreatic tumor or cyst (unspecified) (2/3).

      GroupNotHPOs: HP:0005584 (Renal cell carcinoma)

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: 8634692

      SupplementalData: N/A

      Variant: p.N78T (c.233A>C)

      LegacyVariant: c.446A>C

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223168, https://www.ncbi.nlm.nih.gov/clinvar/variation/223168/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: N78T (c.233A>C)

      MultipleGeneVariants: N/A

    1. 93VHLE3c.500G>Ap.R167QPP7987306M1013936NoPCC6.5Yes

      PatientID: 93

      KindredID: 93

      Case: M, 36Y0M, Chinese (East Asian)

      DiseaseAssertion: Pheochromocytoma

      FamilyInfo: Positive family history.

      CasePresentingHPOs: Pheochromocytoma (HP:0002666)

      CaseHPOFreeText: Pheochromocytoma, with a biggest tumor size of 6.5cm. Age of onset at 10Y and diagnosis at 13Y. 36 years later they were confirmed with a genetic mutation

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: MAX, NF1, RET, SDHA, SDHB, SDHC, SDHD, SDHAF2, TMEM127

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167Q (c.500G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2216, https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: N/A

      gnomAD: 0.000003977, https://gnomad.broadinstitute.org/variant/3-10191507-G-A

      VariantEvidence: Described as a pathogenic variant, according to the ACMG guidelines at the time of publication.

      MutationType: missense_variant;transition

      CivicName: R167Q (c.500G>A)

      MultipleGeneVariants: N/A

    1. PatientID: N/A

      KindredID: 7

      Case: F, 62Y0M, Chinese

      DiseaseAssertion: hemangioblastoma

      FamilyInfo: Patient had family history of VHL disease. Patient’s nephew presented with renal cell carcinoma at 30, patient's sister/brother presented with a hemangioblastoma at age 39 and then retinal capillary hemangioma, renal cell carcinoma, and abnormality of the pancreas at age 57. Patient’s brother presented with retinal hemangioma at age 25 and renal cell carcinoma at age 54. All three of these family members were genetically tested and possessed the same mutation. Patient’s other brother presented with a hemangioblastoma at age 35 but died before diagnosis and genetic testing.

      CohortInfo: 16 Chinese patients clinically diagnosed with VHL disease.

      CasePresentingHPOs: HP:0010797 (hemangioblastoma)

      CaseHPOFreeText: The patient presented with hemangioblastoma at age 40

      CaseNotHPOs: HP:0009711, HP:0002666, HP:0030393, HP:0006770, HP:0001732 (retinal capillary hemangioma, pheochromocytoma, endolymphatic sac tumor, clear cell renal cell carcinoma, abnormality of the pancreas)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.L178R (c.533T>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 625261, https://www.ncbi.nlm.nih.gov/clinvar/variation/625261/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: L178R (c.533T>G)

      MultipleGeneVariants: N/A

    1. Mother3c.499C > T, p.Arg167TrpAsy––––––

      PatientID: None

      KindredID: 12

      Case: F, Age Unknown, Chinese

      DiseaseAssertion:Asymptomatic

      FamilyInfo: Positive. The patient's son was confirmed to have the genetic mutation and presented with pheochromocytoma, renal cell carcinoma, and abnormality of the pancreas.

      CasePresentingHPOs: N/A

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0010797, HP:0009711, HP:0002666, HP:0005584, HP:0001732 (hemangioblastoma, retinal capillary hemangioma, pheochromocytoma, renal cell carcinoma, abnormality of the pancreas)

      CaseNotHPOFreeText: The patient did not present with epididymal or ovarian cystadenomas.

      CasePreviousTesting: N/A

      PreviouslyPublished:29330336

      SupplementalData: N/A

      Variant: p.R167W (c.499C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2218 https://www.ncbi.nlm.nih.gov/clinvar/variation/2218/

      CAID: N/A

      gnomAD: 0.000007954 https://gnomad.broadinstitute.org/variant/3-10191506-C-T

      VariantEvidence: N/A

      MutationType: missense_variant; transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    2. Family 13+3c.464-468delTGTATYes1–343434––

      PatientID: None

      KindredID: 13

      Case: Sex Unknown, 34Y0M, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: Positive. The patient's mother had a hemangioblastoma at age 40 but was not genetically confirmed to have the mutation.

      CasePresentingHPOs: HP:0005584, HP:0001732, HP:0009711(renal cell carcinoma, abnormality of the pancreas, retinal capillary hemangioma)

      CaseHPOFreeText: The patient presented with cysts or tumours of the pancreas, renal cell carcinoma, and retinal angioma at age 34.

      CaseNotHPOs: HP:0010797, HP:0002666 (hemangioblastoma, pheochromocytoma)

      CaseNotHPOFreeText: The patient did not present with epididymal or ovarian cystadenomas.

      CasePreviousTesting: N/A

      PreviouslyPublished:29330336

      SupplementalData: N/A

      Variant:p.V155Dfs*17(c.464_468delTGTAT)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA2499306942 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/by_caid?caid=CA2499306942&fbclid=IwAR1k8AeHEqS_dwb3ClO4W8AWqBs2tf0malEPJ2nbl-bl7ZkgNC3jeWbaDds

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: minus_2_frameshift_variant

      CivicName:V155fs(c.464_468delTGTAT)

      MultipleGeneVariants: N/A

    3. Family 14+3C.481C > T, p.Arg161StopNo129–2933–– Father∗Unknown42–––––

      PatientID: None

      KindredID: 14

      Case: Sex Unknown, 33Y0M, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: The patient's father had a hemangioblastoma of the central nervous system at age 42. He was deceased before diagnosis could occur.

      CasePresentingHPOs: HP:0010797, HP:0005584, HP:0001732 (hemangioblastoma, renal cell carcinoma, abnormality of the pancreas)

      CaseHPOFreeText: The patient presented with hemangioblastoma and renal cell carcinoma at age 29. At age 33, the patient presented with pancreatic cysts or tumours.

      CaseNotHPOs: HP:0009711, HP:0002666, HP:0009715 (retinal capillary hemangioma, pheochromocytoma, Papillary cystadenoma of the epididymis)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished:29330336

      SupplementalData: N/A

      Variant: p.R161* (c.481C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2217 https://www.ncbi.nlm.nih.gov/clinvar/variation/2217/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: stop_gained

      CivicName: R161*(c.481C>T)

      MultipleGeneVariants: N/A

    4. Family 15−3c.481C > T, p.Arg161StopNo139–3939––

      PatientID: None

      KindredID: 15

      Case: Sex Unknown, 39Y0M, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: None

      CasePresentingHPOs: HP:0010797, HP:0005584, HP:0001732 (hemangioblastoma, renal cell carcinoma, abnormality of the pancreas)

      CaseHPOFreeText: The patient presented with hemangioblastoma, renal cell carcinoma, and pancreatic cysts or tumours at age 39.

      CaseNotHPOs: HP:0009711, HP:0002666, HP:0009715 (retinal capillary hemangioma, pheochromocytoma, Papillary cystadenoma of the epididymis)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 29330336

      SupplementalData: N/A

      Variant: p.R161* (c.481C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2217 https://www.ncbi.nlm.nih.gov/clinvar/variation/2217/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: stop_gained

      CivicName: R161*(c.481C>T)

      MultipleGeneVariants: N/A

    5. Nephew 32c.349T > G, p.Trp117Gly1313––––

      PatientID: None

      KindredID: 6

      Case: M, 13Y0M, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: Positive. Patient's aunt/uncle was the proband and had the mutation c.349T>G presenting with renal cell carcinoma, retinal angioma, hemangioblastoma, abnormality of the pancreas, and epididymal or ovarian cystadenoma. Many other members of the family had VHL associated phenotypes and three others were confirmed to have this genetic mutation. Additional familial phenotypes can be found in Table 1.

      CasePresentingHPOs: HP:0010797, HP:0009711 (hemangioblastoma, retinal capillary hemangioma)

      CaseHPOFreeText: The patient presented with hemangioblastoma and retinal angioma at age 13.

      CaseNotHPOs: HP:0002666, , HP:0005584, HP:0001732, (pheochromocytoma, renal cell carcinoma, abnormality of the pancreas)

      CaseNotHPOFreeText: The patient did not have a cystadenoma of the epididymis.

      CasePreviousTesting: N/A

      PreviouslyPublished: 29330336

      Variant: p.W117G (c.349T>G)

      LegacyVariant:N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 862346 https://www.ncbi.nlm.nih.gov/clinvar/variation/862346/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName:W117G (c.349T>G)

      MultipleGeneVariants: N/A

    Tags

    Annotators

    URL

    1. PatientID: 1

      KindredID: V5PG

      Case: Age unknown, sex unknown, Italian

      DiseaseAssertion: assumed VHL

      FamilyInfo: 2 twin patients belonging to family V5PG were examined. Both with hemangioblastomas and 1 also with retinal hemangioblastomas. Sequencing of patients’ DNA and paternity and monozygosity of the 2 twins was ascertained, revealed a de novo missense mutation.

      CasePresentingHPOs: HP:0009711; HP:0010797 (retinal capillary hemangioblastoma, hemangioblastoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0005584; HP:0002666 (renal cell carcinoma, pheochromocytoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.N78S (c.233A>G)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: 446 (78) A>G (N78S)

      ClinVarID: 93326 https://www.ncbi.nlm.nih.gov/clinvar/variation/93326/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transition

      CivicName: N78S (c.233A>G)

      MultipleGeneVariants: N/A

    1. c.99_100InsA*

      PatientID: None (XI)

      KindredID: 7

      Case: Sex and Ethnicity unknown; Age >18Y0M

      DiseaseAssertion: assumed VHL

      FamilyInfo: Total of 13 family members belonging to Family 7 (see Table 2; or follow KindredID 7)

      CasePresentingHPOs: HP:0010797; HP:0000107; HP:0001737; HP:0005584 (hemangioblastoma, renal cyst, pancreatic cysts, renal cell carcinoma)

      CaseHPOFreeText: initially thought to be asymptomatic, first screening showed 1 HB; 5 year follow up showed +RC, PC, RCC

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 16572651

      SupplementalData: N/A

      Variant: p.G34Rfs*98 (c.99_100insA)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: N/A

      CAID: CA2018007671 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/by_caid?caid=CA2018007671

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: plus_1_frameshift_variant

      CivicName: G34Rfs (c.99_100insA)

      MultipleGeneVariants: N/A

    2. 6c.99_100InsA*>18YAffected1 Hb, Bilat RASame

      PatientID: None (I)

      KindredID: 6

      Case: Sex and Ethnicity unknown; Age >18Y0M

      DiseaseAssertion: assumed VHL

      FamilyInfo: Total of 6 family members belonging to Family 6 (see Table 2; or follow KindredID 6)

      CasePresentingHPOs: HP:0010797; HP:0009711(hemangioblastoma, retinal capillary hemangioma)

      CaseHPOFreeText: 1 HB and bilateral RA at first and final screenings

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 16572651

      SupplementalData: N/A

      Variant: p.G34Rfs*98 (c.99_100insA)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: N/A

      CAID: CA2018007671 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/by_caid?caid=CA2018007671

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: plus_1_frameshift_variant

      CivicName: G34Rfs (c.99_100insA)

      MultipleGeneVariants: N/A

    3. p.F76del

      PatientID: None (IV)

      KindredID: 1

      Case: Sex and Ethnicity unknown; Age <18Y0M

      DiseaseAssertion: asymptomatic

      FamilyInfo: Total of 4 family members belonging to Family 1 (see Table 2; or follow KindredID 1)

      CasePresentingHPOs: None

      CaseHPOFreeText: asymptomatic at first and final (5 year follow up) screening

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 16572651

      SupplementalData: N/A

      Variant: p.F76del (c.227_229del)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 223166 https://www.ncbi.nlm.nih.gov/clinvar/variation/223166/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: inframe_deletion

      CivicName: p.F76del

      MultipleGeneVariants: N/A

    1. Germline mutations were identified in 300/469 (63%) of the families tested; 137 distinct intragenic germline mutations were detected.

      GroupID/ KindredID: 30CE

      DiseaseAssertion: VHL

      GroupSize: N/A

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: This germline familial VHL mutation was taken from the Munich, university of Freiburg and Institute of Pathology database, with an unknown number of members belonging to Family ID 30CE. Table 3 lists all ID and mutations associated with individuals possessing renal carcinoma, without pheochromocytoma.

      GroupPresentingHPOs: HP:0005548 (renal cell carcinoma)

      GroupHPOFreeText: Described as a VHL family with RCC, without pheochromocytoma. Other phenotypes are likely, however not detailed in this publication

      GroupNotHPOs: HP:0002666 (pheochromocytoma)

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.Q132* (c.394C>T)

      LegacyVariant: p.Q132* (c.607C>T)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 288749 https://www.ncbi.nlm.nih.gov/clinvar/variation/288749/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: This paper declares this variant is predicted to compromise the Elongin-binding site by truncating the protein.

      MutationType: stop_gained

      CivicName: Q132* (c.394C>T)

      MultipleGeneVariants: N/A

    2. Germline mutations were identified in 300/469 (63%) of the families tested; 137 distinct intragenic germline mutations were detected.

      GroupID/ KindredID: 8BMS

      DiseaseAssertion: VHL

      GroupSize: N/A

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: This germline familial VHL mutation was taken from the Bristol Myers Squibb database, with an unknown number of members belonging to family ID: Family 8BMS. Table 5 lists all ID and mutations associated with individuals without detailed clinical information.

      GroupPresentingHPOs: N/A

      GroupHPOFreeText: Described as multiple VHL families without detailed clinical information. Other phenotypes are possible, however not detailed here.

      GroupNotHPOs: N/A

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.L135* (c.404T>A)

      LegacyVariant: p.L135* (c.617T>A)

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA351754021 https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.404T>A

      gnomAD: N/A

      VariantEvidence: This paper declares this variant is predicted to compromise the Elongin-binding site by truncating the protein.

      MutationType: stop_gained

      CivicName: L135* (c.404T>A)

      MultipleGeneVariants: N/A

    3. Germline mutations were identified in 300/469 (63%) of the families tested; 137 distinct intragenic germline mutations were detected.

      GroupID/ KindredID: 48CE

      DiseaseAssertion: VHL

      GroupSize: N/A

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: This germline familial VHL mutation was taken from the Munich, university of Freiburg and Institute of Pathology database, with an unknown number of members belonging to Family ID 48CE. Table 3 lists all ID and mutations associated with individuals possessing renal carcinoma, without pheochromocytoma.

      GroupPresentingHPOs: HP:0005548 (renal cell carcinoma)

      GroupHPOFreeText: Described as a VHL family with RCC, without pheochromocytoma. Other phenotypes are likely, however not detailed in this publication

      GroupNotHPOs: HP:0002666 (pheochromocytoma)

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: (c.464-1G>A)

      LegacyVariant: Splice (c.677-1G>A)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 43603 https://www.ncbi.nlm.nih.gov/clinvar/variation/43603/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: This paper declares this variant is predicted to compromise the Elongin-binding site by truncating the protein.

      MutationType: splice_site_variant

      CivicName: Splice Site (c.464-1G>A)

      MultipleGeneVariants: N/A

    1. PatientID: 3

      KindredID: II

      Case: Sex Unknown, Age Unknown, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: None

      CasePresentingHPOs: HP:0010797 (hemangioblastoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: c.340+1G>A

      CaseProblemVariantFreeText: N/A

      LegacyVariant: c.553+1G>A

      ClinVarID: 223190 https://www.ncbi.nlm.nih.gov/clinvar/variation/223190/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: Mutation was predicted to compromise the elongin-binding region. Refer to table 3 for LOH information on VHL and ZAC1.

      MutationType: splice_site_variant

      CivicName: Splice Site (c.340+1G>A)

      MultipleGeneVariants: N/A

    1. proband (F2-II-11)

      PatientID: proband (F2-II-11)

      KindredID: 2

      Case: F, 45Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: This patients son (F2-III-9) was a 9-year old male patient with a right adrenal pheochromocytoma. The son later presented with a left adrenal pheochromocytoma at 17-years-old. The patients niece (F2-III-4) was a 33 year old female with bilateral pheochromocytoma. The patients brother (F2-II-3) succumbed to hypertensive intracerebral hemorrhage 21 years prior to this study.

      CasePresentingHPOs: HP:0006748 (adrenal pheochromocytoma)

      CaseHPOFreeText: presented with paroxysmal hypertension (180–210/100–120 mmHg), headache, palpitations and hyperhidrosis in 1985 at the age of 18. US scans showed an isolated mass in the right adrenal gland.

      CaseNotHPOs: HP:0005584, HP:0000107, HP:0010797 (renal cell carcinoma, renal cyst, hemangioblastoma)

      CaseNotHPOFreeText: no other systematic tumors, including CNS hemangioblastomas, renal cysts or renal cell carcinoma, were identified

      CasePreviousTesting: RET

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R161Q (c.482G>A)

      LegacyVariant: N/A

      ClinVar: 182983 https://www.ncbi.nlm.nih.gov/clinvar/variation/182983/

      CAID: CA020413

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R161Q (c.482G>A)

      MultipleGeneVariants: N/A

    2. F1-III-1

      PatientID: F1-III-1

      KindredID: F1

      Case: M, 5Y0M, Ethnicity Unknown

      DiseaseAssertion: asymptomatic

      FamilyInfo: This patients father (F1-II-1) was a 31-year old male patient with pheochromocytoma, renal cell carcinoma, and epididymal papillary adenoma. This patients uncle (F1-II-3) was a 30 year-old who presented with multiple right renal masses, epididymal nodules and multiple pancreatic cysts. Histopathological analysis revealed right renal cell carcinoma with hemorrhagic cyst

      CasePresentingHPOs: None

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: The patient is asymptomatic

      CasePreviousTesting: RET

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.N78S (c.233A>G)

      LegacyVariant: N/A

      ClinVar: 93326 https://www.ncbi.nlm.nih.gov/clinvar/variation/93326/

      CAID: CA020131

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: N78S (c.233A>G)

      MultipleGeneVariants: N/A

    1. KindredID: 152

      GroupID: 47

      GroupSize: Unknown

      GroupNumberMales: Unknown

      GroupNumberFemales: Unknown

      GroupEthnicity: Unknown

      GroupAgeRange: Unknown

      DiseaseAssertion: VHL

      GroupInfo: Kindred no.152 describes a family where at least one member (unspecified how many) had this mutation in the VHL gene. Family history indicates that pheochromocytoma is not present in this kindred.

      GroupPresentingHPOs: N/A

      GroupHPOFreeText: N/A

      GroupNotHPOs: HP:0002666 (Pheochromocytoma)

      GroupNotHPOFreeText: Pheochromocytoma was not observed in any members of the affected patient(s)' family (VHL type 1).

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167Q (c.500G>A)

      LegacyVariant: c.713G>A (p.R238Q)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2216, https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: N/A

      gnomAD: 0.000003977, https://gnomad.broadinstitute.org/variant/3-10191507-G-A?dataset=gnomad_r2_1

      VariantEvidence:

      MutationType: missense_variant;transition

      CivicName: R167Q (c.500G>A)

      MultipleGeneVariants: N/A

    2. KindredID: 136

      GroupID: 21

      GroupSize: Unknown

      GroupNumberMales: Unknown

      GroupNumberFemales: Unknown

      GroupEthnicity: Unknown

      GroupAgeRange: Unknown

      DiseaseAssertion: VHL

      GroupInfo: Kindred no.136 describes a family where at least one member (unspecified how many) had this mutation in the VHL gene. Family history indicates that pheochromocytoma is not present in this kindred.

      GroupPresentingHPOs: N/A

      GroupHPOFreeText: N/A

      GroupNotHPOs: HP:0002666 (Pheochromocytoma)

      GroupNotHPOFreeText: Pheochromocytoma was not observed in any members of the affected patient(s)' family (VHL type 1).

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R113* (c.337C>T)

      LegacyVariant: c.550C>T (p.R184*)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 220414, https://www.ncbi.nlm.nih.gov/clinvar/variation/220414/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: stop_gained

      CivicName: R113* (c.337C>T)

      MultipleGeneVariants: N/A

    3. KindredID: 79

      GroupID: 51

      GroupSize: Unknown

      GroupNumberMales: Unknown

      GroupNumberFemales: Unknown

      GroupEthnicity: Unknown

      GroupAgeRange: Unknown

      DiseaseAssertion: VHL

      GroupInfo: Kindred no. 79 describes a family where at least one member (unspecified how many) had this mutation in the VHL gene. Pheochromocytoma was not observed in this kindred indicating VHL disease type 1.

      GroupPresentingHPOs: N/A

      GroupHPOFreeText: N/A

      GroupNotHPOs: HP:0002666 (Pheochromocytoma)

      GroupNotHPOFreeText: Pheochromocytoma was not observed in this kindred indicating VHL disease type 1.

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.I180V (c.538A>G)

      LegacyVariant: p.I251V (c.751A>G)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 161401, https://www.ncbi.nlm.nih.gov/clinvar/variation/161401/

      CAID: N/A

      gnomAD: 0.00001061, https://gnomad.broadinstitute.org/variant/3-10191545-A-G?dataset=gnomad_r2_1

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: I180V (c.538A>G)

      MultipleGeneVariants: N/A

    4. KindredID: 11

      GroupID: 52

      GroupSize: Unknown

      GroupNumberMales: Unknown

      GroupNumberFemales: Unknown

      GroupEthnicity: Unknown

      GroupAgeRange: Unknown

      DiseaseAssertion: VHL

      GroupInfo: Kindred no. 11 describes a family where at least one member (unspecified how many) had this mutation in the VHL gene. Pheochromocytoma was not observed in this kindred indicating VHL disease type 1.

      GroupPresentingHPOs: N/A

      GroupHPOFreeText: N/A

      GroupNotHPOs: HP:0002666 (Pheochromocytoma)

      GroupNotHPOFreeText: Pheochromocytoma was not observed in this kindred indicating VHL disease type 1.

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.L184R (c.551T>G)

      LegacyVariant: p.L255R (c.764T>G)

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA351756355, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.551T%3EG

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: L184R (c.551T>G)

      MultipleGeneVariants: N/A

    5. KindredID: 49

      GroupID: 31

      GroupSize: Unknown

      GroupNumberMales: Unknown

      GroupNumberFemales: Unknown

      GroupEthnicity: Unknown

      GroupAgeRange: Unknown

      DiseaseAssertion: VHL

      GroupInfo: Kindred no. 49 describes a family where at least one member (unspecified how many) had this mutation in the VHL gene. Pheochromocytoma was observed in this kindred indicating VHL disease type 2.

      GroupPresentingHPOs: HP:0002666 (Pheochromocytoma)

      GroupHPOFreeText: Pheochromocytoma was observed in this kindred indicating VHL disease type 2.

      GroupNotHPOs: N/A

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.P154L (c.461C>T)

      LegacyVariant: p.P225L (c.674C>T)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 649525, https://www.ncbi.nlm.nih.gov/clinvar/variation/649525/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: P154L (c.461C>T)

      MultipleGeneVariants: N/A

    1. PatientID: 161

      KindredID: 62

      Case: F, 41Y0M, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: The patient's daughter presented with a CNS hemangioblastoma and multiple pancreatic cysts and tumors at the age of 14.

      CasePresentingHPOs: HP:0005584; HP:0002666; HP:0010797; HP:0001737; HP:0001732 (Renal cell carcinoma, Pheochromocytoma, Hemangioblastoma, Pancreatic cysts, Abnormality of the pancreas)

      CaseHPOFreeText: The proband presented with a CNS hemangioblastoma at age 28, and a renal cell carcinoma, multiple pancreatic cysts/tumors, and a pheochromocytoma at age 41.

      CaseNotHPOs: HP:0009711 (Retinal capillary hemangioma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: This cohort was also published in a later paper by Wang et al., 2018 (PMID: 29330336).

      SupplementalData: Patient and family genotype-phenotype information is listed in Supplementary Table 1.

      Variant: p.R161* (c.481C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2217, https://www.ncbi.nlm.nih.gov/clinvar/variation/2217/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R161* (c.481C>T)

      MultipleGeneVariants: N/A

    2. PatientID: 195

      KindredID: 75

      Case: M, 32Y0M, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0002666 (Pheochromocytoma)

      CaseHPOFreeText: The patient presented with a pheochromocytoma at age 32.

      CaseNotHPOs: HP:0005584; HP:0009711; HP:0001737; HP:0001732; HP:0010797 (Renal cell carcinoma, Retinal capillary hemangioma, Pancreatic cysts, Abnormality of the pancreas, CNS hemangioblastoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: This patient was also published in a later paper by Wang et al., 2018 (PMID: 29330336).

      SupplementalData: Patient and family genotype-phenotype information is listed in Supplementary Table 1.

      Variant: p.R167W (c.499C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2218; https://www.ncbi.nlm.nih.gov/clinvar/variation/2218/

      CAID: N/A

      gnomAD: 0.000007954; https://gnomad.broadinstitute.org/variant/3-10191506-C-T?dataset=gnomad_r2_1

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    3. PatientID: 179

      KindredID: 70

      Case: F, 44Y0M, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: The proband (patient 179) had a daughter (patient 180) who was confirmed with the mutation, and present4ed with a retinal angioma and multiple pancreatic cysts/tumors at 17. The proband also has three family members who were unconfirmed with the mutation (passed away before diagnosis): patient 181 (proband's brother) had a CNS hemangioblastoma at 17, patient 182 (proband's father) had a CNS hemangioblastoma at 49, renal cell carcinoma and multiple pancreatic cysts/tumors at 56, and patient 183 (proband's uncle) had a CNS hemangioblastoma at 32 and a retinal angioma at 30.

      CasePresentingHPOs: HP:0001737; HP:0001732; HP:0005584 (Pancreatic cysts, Abnormality of the pancreas, Renal cell carcinoma)

      CaseHPOFreeText: The proband presented with a renal cell carcinoma at 44, and multiple pancreatic cysts/tumors at 28.

      CaseNotHPOs: HP:0010797; HP:0002666; HP:0009711 (Hemangioblastoma, Pheochromocytoma, Retinal capillary hemangioma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: This patient was also published in a later paper by Wang et al., 2018 (PMID: 29330336).

      SupplementalData: Patient and family genotype-phenotype information is listed in Supplementary Table 1.

      Variant: p.C162* (c.486C>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223226; https://www.ncbi.nlm.nih.gov/clinvar/variation/223226/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: C162* (c.486C>A)

      MultipleGeneVariants: N/A

    4. PatientID: 150

      KindredID: 55

      Case: M, 17Y0M, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: The proband (patient 150) has a father (patient 151) who was also confirmed with this germline mutation, and presented at 22 with a CNS hemangioblastoma and 28 with renal cell carcinoma. The proband also has a grandmother (patient 153) who presented with a CNS hemangioblastoma at 43, but who passed away before diagnosis/ genetic testing.

      CasePresentingHPOs: HP:0010797; HP:0001737; HP:0001732 (Hemangioblastoma, Pancreatic cysts, Abnormality of the pancreas)

      CaseHPOFreeText: The proband presented at age 14 with a CNS hemangioblastoma, and age 17 with multiple pancretic cysts/tumors.

      CaseNotHPOs: HP:0009711; HP:0002666; HP:0005584 (Retinal capillary hemangioma, Pheochromocytoma, Renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: This patient was also published in a later paper by Wang et al., 2018 (PMID: 29330336).

      SupplementalData: Patient and family genotype-phenotype information is listed in Supplementary Table 1.

      Variant: p.I151S (c.452T>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223215, https://www.ncbi.nlm.nih.gov/clinvar/variation/223215/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: I151S (c.452T>G)

      MultipleGeneVariants: N/A

    5. PatientID: 215

      KindredID: 80

      Case: F, 14Y0M, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: The patient's brother (the proband) and nephew both had the same mutation. Their brother had a CNS hemangioblastoma at 26. Their nephew had a retinal angioma at 26, and a pheochromocytoma at 31. Their mother had renal cell carcinoma at 56, however no genotyping information is available for her.

      CasePresentingHPOs: ; HP:0002666 (Pheochromocytoma)

      CaseHPOFreeText: The patient presented with a pheochromocytoma at age 14.

      CaseNotHPOs: HP:0001737; HP:0001732; HP:0005584; HP:0010797; HP:0009711 (Pancreatic cysts, Abnormality of the pancreas, Renal cell carcinoma, Hemangioblastoma, Retinal Capillary Hemangioma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: This cohort was also published in a later paper by Wang et al., 2018 (PMID: 29330336).

      SupplementalData: Patient and family genotype-phenotype information is listed in Supplementary Table 1.

      Variant: p.R167Q (c.500G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2216, https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167Q (c.500G>A)

      MultipleGeneVariants: N/A

    6. PatientID: 48

      KindredID: 15

      Case: F, 19Y0M, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: Proband (patient 42) has four family members confirmed with the mutation, and two family members who passed away before diagnosis/ genetic confirmation of the mutation. Family members confirmed with the mutation include: the proband's daughter (patient 43) who had retinal angioma (age 17), renal cell carcinoma (21), multiple pancreatic cysts/tumors (18), and a pheochromocytoma (19). His son (patient 44) who was asymptomatic at the time of assessment. His niece 1 (patient 47) who had a retinal angioma and renal cell carcinoma (22). And his niece 2 (patient 28) who had a CNS hemangioblastoma (19) and a retinal angioma (18). Family members who passed away before diagnosis and were not genetically confirmed include: his sister (oatient 45) who had a CNS hemangioblastoma (22), and his brother (patient 46) who had a retinal angioma and renal cell carcinoma at 52.

      CasePresentingHPOs: HP:0009711; HP:0010797 (Retinal capillary hemangioma, Hemangioblastoma)

      CaseHPOFreeText: The proband's niece 2 (patient 48) had a retinal angioma at 18 and a CNS hemangioblastoma at 19.

      CaseNotHPOs: HP:0001737; HP:0001732; HP:0002666; HP:0005584 (Pancreatic cysts, Abnormality of the pancreas, Pheochromocytoma, Renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: This patient was also published in a later paper by Wang et al., 2018 (PMID: 29330336).

      SupplementalData: Patient and family genotype-phenotype information is listed in Supplementary Table 1.

      Variant: p.S80Tfs*77 (c.239_245del)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA913189246; https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.239_245del

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: minus_1_frameshift_variant

      CivicName: S80fs (c.239_245del)

      MultipleGeneVariants: N/A

    7. PatientID: 113

      KindredID: 41

      Case: M, 36Y0M, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0010797; HP:0005584; HP:0001737; HP:0001732 (Hemangioblastoma, Renal cell carcinoma, Pancreatic cysts, Abnormality of the pancreas)

      CaseHPOFreeText: The proband presented with a CNS hemangioblastoma at 26, and at age 36 presented with renal cell carcinoma and multiple pancreatic cysts/tumors.

      CaseNotHPOs: HP:0009711; HP:0002666 (Retinal capillary hemangioma, Pheochromocytoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: This cohort was also published in an earlier paper by Wang et al., 2014 (PMID 24581539) as well as a later paper by Wang et al., 2018 (PMID: 29330336).

      SupplementalData: Patient and family genotype-phenotype information is listed in Supplementary Table 1.

      Variant: p.H115Tfs*44 (c.343del)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA432421784, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.343delC

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: minus_1_frameshift_variant

      CivicName: H115FS (c.343delC)

      MultipleGeneVariants: N/A

    8. PatientID: 57

      KindredID: 20

      Case: F, 18Y0M, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: The patient had a CNS hemangioblastoma at age 18. Their father (listed in this paper as the proband) presented with a retinal angioma at 32, a CNS hemangioblastoma at 34, and renal cell carcinoma as well as multiple pancreatic cysts/tumors at 38. Their father passed away before genetic testing could be confirmed.

      CasePresentingHPOs: HP:0010797 (CNS Hemangioblastoma)

      CaseHPOFreeText: The proband had a CNS hemangioblastoma at age 18.

      CaseNotHPOs: HP:0009711; HP:0002666; HP:0005584; HP:0001737; HP:0001732 (Retinal capillary hemangioma, Pheochromocytoma, Renal cell carcinoma, Pancreatic cysts, Abnormality of the pancreas)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: This cohort was also published in a later paper by Wang et al., 2018 (PMID: 29330336).

      SupplementalData: Patient and family genotype-phenotype information is listed in Supplementary Table 1.

      Variant: p.P86L (c.257C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 182977, https://www.ncbi.nlm.nih.gov/clinvar/variation/182977/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: P86L (c.257C>T)

      MultipleGeneVariants: N/A

    Tags

    Annotators

    URL

    1. 21 missense mutations

      PatientID: 25

      KindredID: 1

      Case: Sex unknown, Age unknown, French

      DiseaseAssertion: VHL

      FamilyInfo: One other member belonging to family 1 was mentioned (Patient: 33). This patient was found to have a pancreatic endocrine tumour, pheochromocytoma and renal cell carcinoma.

      CasePresentingHPOs: HP:0030405; HP:0002666 (pancreatic endocrine tumour, pheochromocytoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0005584 (renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167W (c.499C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2218 https://www.ncbi.nlm.nih.gov/clinvar/variation/2218/

      CAID: N/A

      gnomAD: 0.000007954 https://gnomad.broadinstitute.org/variant/3-10191506-C-T

      VariantEvidence:N/A

      MutationType: missense_variant;transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    2. 5 nonsense mutations

      PatientID: 11

      KindredID: 63

      Case: Sex unknown, Age unknown, French

      DiseaseAssertion: VHL

      FamilyInfo: One other member belonging to family 63 was mentioned (Patient: 19). This patient was found to have pheochromocytoma, renal cell carcinoma and pancreatic endocrine tumour.

      CasePresentingHPOs: HP:0030405 (pancreatic endocrine tumour)

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0002666; HP:0005584 (pheochromocytoma, renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.Y175* (c.525C>G)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 182974 https://www.ncbi.nlm.nih.gov/clinvar/variation/182974/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: stop_gained

      CivicName: Y175* (c.525C>G)

      MultipleGeneVariants: N/A

    3. 21 missense mutations, 5 nonsense mutations

      PatientID: 34

      KindredID: 94

      Case: Sex unknown, Age unknown, French

      DiseaseAssertion: VHL

      FamilyInfo: None

      CasePresentingHPOs: HP:0005584; HP:0030405 (renal cell carcinoma, pancreatic endocrine tumour)

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0002666 (pheochromocytoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.F136S (c.407T>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 496064 https://www.ncbi.nlm.nih.gov/clinvar/variation/496064/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: F136S (c.407T>C)

      MultipleGeneVariants: N/A

    1. The cytosine replaced the 716th guanine on four patients and three carriers of virulence gene from the first family, which made the 168th serine replaced by threonine.

      PatientID: III-11

      KindredID: 1

      Case: M, Age Unknown, Chinese

      DiseaseAssertion: CNS Hemangioblastoma

      FamilyInfo: The transversion mutation was found and confirmed in 4 patients (II-2, III-2, III-6, III-10, III-11) and 3 carriers (III-3, III-9, III-14). At least 5 additional members had VHL phenotypes, but had all passed away before genetic testing. Could be assumed and deduced their genetics due to pedigree showing future linear relations of this mutation all in generation III.

      CasePresentingHPOs: HP:0010797 (hemangioblastoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.S168T (c.503G>C)

      LegacyVariant: p.S168T (c.716G>C)

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA351756183 https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.503G%3EC

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: S168T (c.503G>C)

      MultipleGeneVariants: N/A

    2. The cytosine replaced the 716th guanine on four patients and three carriers of virulence gene from the first family, which made the 168th serine replaced by threonine.

      PatientID: III-10

      KindredID: 1

      Case: M, Age Unknown, Chinese

      DiseaseAssertion: CNS Hemangioblastoma

      FamilyInfo: The transversion mutation was found and confirmed in 4 patients (II-2, III-2, III-6, III-10, III-11) and 3 carriers (III-3, III-9, III-14). At least 5 additional members had VHL phenotypes, but had all passed away before genetic testing. Could be assumed and deduced their genetics due to pedigree showing future linear relations of this mutation all in generation III.

      CasePresentingHPOs: HP:0010797 (hemangioblastoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.S168T (c.503G>C)

      LegacyVariant: p.S168T (c.716G>C)

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA351756183 https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.503G%3EC

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: S168T (c.503G>C)

      MultipleGeneVariants: N/A

    1. PatientID: None (Proband)

      KindredID: 1

      Case: M, 49Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: This patient had two children with the same genetic mutation. Son presented with bilateral pheochromocytoma (13Y) and endolymphatic sac tumor (19Y). Daughter was asymptomatic.

      CasePresentingHPOs: HP:0005584; HP:0002666; HP:0001945; HP:0000790 (Renal cell carcinoma, Pheochromocytoma, Fever, Hematuria)

      CaseHPOFreeText: A 48Y man presented with loin pain, pyrexia and haematuria. He was initially thought to have pyelonephritis but a renal ultrasound scan showed a right renal mass. The CT scan and the MRI confirmed the finding of a large right renal mass invading the renal vein and also identified bilateral adrenal masses. hoarse voice due to a mass impinging on the laryngeal nerves. A diagnosis of VHL presenting with a metastatic right RCC and bilateral pheochromocytomas was made. Despite chemotherapy for the metastatic RCC, he died 1 year after diagnosis.

      CaseNotHPOs: HP:0000822 (Hypertension)

      CaseNotHPOFreeText: He was normotensive at 48Y.

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167W (c.499C>T)

      LegacyVariant: c.712C>T

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2218, https://www.ncbi.nlm.nih.gov/clinvar/variation/2218/

      CAID: N/A

      gnomAD: 0.000007954, https://gnomad.broadinstitute.org/variant/3-10191506-C-T

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    1. 25Fc.499C>T3Missensep.Arg167Trp12P, mu

      PatientID: 25

      KindredID: 4

      Case: F, 12Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Four members of this family, IDs #22 (8Y, F), #23 (25Y, M), #24 (14Y, M), #25 (12Y, M), were found to have the same variant and pheochromocytoma.

      CohortInfo: Thirty-one subjects were included in this study consisting of 16 SDHB mutation carriers and 15 subjects with VHL disease followed-up between 1975 and 2008. Subjects were included in the study if they had complete imaging from the base of the skull to the pelvis and measurements of urinary or plasma catecholamines. The case-notes and hospital databases were examined for the required data, which included: age at first symptom/diagnosis, family history, disease penetrance, site and size of tumor, symptoms, secretion of catecholamines, multifocal disease, metastatic disease, treatment and disease outcome.

      CasePresentingHPOs: HP:0002666 (Pheochromocytoma)

      CaseHPOFreeText: Multifocal pheochromocytoma. 12Y at VHL diagnosis.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167W (c.499C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2218, https://www.ncbi.nlm.nih.gov/clinvar/variation/2218/

      CAID: N/A

      gnomAD: 0.000007954, https://gnomad.broadinstitute.org/variant/3-10191506-C-T

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    2. 18aMc.555C>G3Nonsensep.Tyr185X30APRetinal angiomas aged 16

      PatientID: 18

      KindredID: A

      Case: M, 30Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CohortInfo: Thirty-one subjects were included in this study consisting of 16 SDHB mutation carriers and 15 subjects with VHL disease followed-up between 1975 and 2008. Subjects were included in the study if they had complete imaging from the base of the skull to the pelvis and measurements of urinary or plasma catecholamines. The case-notes and hospital databases were examined for the required data, which included: age at first symptom/diagnosis, family history, disease penetrance, site and size of tumor, symptoms, secretion of catecholamines, multifocal disease, metastatic disease, treatment and disease outcome.

      CasePresentingHPOs: HP:0009711; HP:0002668 (Retinal capillary hemangioma, Paraganglioma)

      CaseHPOFreeText: Retinal angiomas aged 16Y. 30Y at VHL diagnosis. Abdominal paraganglioma.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.Y185* (c.555C>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223233, https://www.ncbi.nlm.nih.gov/clinvar/variation/223233/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion; stop_gained

      CivicName: Y185* (c.555C>G)

      MultipleGeneVariants: N/A

    1. and one of her daughters (III-1)

      PatientID: III-1

      KindredID: 1

      Case: F, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: Asymptomatic

      FamilyInfo: The patient’s uncle (II-1) was found to harbor the same germline mutation the patient harbored and had bilateral adrenal pheochromocytoma at 14Y. The patient’s aunt (II-2) was found to harbor the same germline mutation the patient harbored and had left adrenal pheochromocytoma at 10Y. The same germline missense mutation was found in the patient’s mother (II-4) who had right adrenal pheochromocytoma. The grandfather was not available for genetic testing because he had died, apparently secondary to a hypertensive crisis. The five other family members which underwent genetic testing did not have the mutation.

      CasePresentingHPOs: N/A

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.V166F (c.496G>T)

      LegacyVariant: N/A

      ClinVarID: 2224, https://www.ncbi.nlm.nih.gov/clinvar/variation/2224/

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: V166F(c.496G>T)

      MultipleGeneVariants: N/A

    1. PatientID: 1

      KindredID: 1

      Case: F, 25Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Her family history was concerning for pheochromocytoma and renal cell carcinoma in her father and a paternal aunt, and a paternal grandmother with history of brain tumor. All of these individuals were deceased. No genetic testing had been performed on any of her affected relatives.

      CasePresentingHPOs: HP:0002666 (pheochromocytoma)

      CaseHPOFreeText: MRI scan ascertained diagnosis of bilateral pheochromocytoma (which preceded by at least 5 years). The pheo and resulting in recurrent episodes of malignant hypertension and intrauterine fetal demise led to the appropriate peripartum diagnosis of VHL syndrome

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167Q (c.500G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2216, https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: N/A

      gnomAD: 0.000003977 https://gnomad.broadinstitute.org/variant/3-10191507-G-A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167Q (c.500G>A)

      MultipleGeneVariants: N/A

    1. 485G>A

      GroupID/ KindredID: 16

      DiseaseAssertion: VHL

      GroupSize: 5

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: Polish

      GroupAgeRange: N/A

      GroupInfo: Familial VHL stated; 5 family members recorded with this mutation.

      GroupPresentingHPOs: HP:0009711; HP:0010797 (retinal capillary hemangioma, hemangioblastoma)

      GroupHPOFreeText: 4 patients were tallied with a retinal capillary hemangiomas, and 4 with cHemangioblastomas. These phenotypes couldn’t be paired as it was not clear with each member.

      GroupNotHPOs: HP:0005584 (renal cell carcinoma)

      GroupNotHPOFreeText: None of the patients were recorded to have RCC

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.C162Y (c.485G>A)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 223225 https://www.ncbi.nlm.nih.gov/clinvar/variation/223225/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transition

      CivicName: C162Y (c.485G>A)

      MultipleGeneVariants: N/A

    2. 233A>G

      GroupID/ KindredID: 8

      Case: Age unknown, Sex unknown, Polish

      DiseaseAssertion: VHL

      FamilyInfo: This mutation (p.N78S (c.233A>G)) was found in Family No. 8 with 1 member affected. Germline de novo mutations confirmed by analysis of parents’ DNA. Germline mutations (of 233A>G substitution) were not detected in DNA extracted from blood leucocytes of the parents.

      CasePresentingHPOs: HP:0009711; HP:0010797 (retinal capillary hemangioma, hemangioblastoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0005584 (renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.N78S (c.233A>G)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 93326 https://www.ncbi.nlm.nih.gov/clinvar/variation/93326/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: N78S (c.233A>G)

      MultipleGeneVariants: N/A

    1. 11 Missense p.(Arg167Gln) 0 2

      GroupID: 11

      DiseaseAssertion: VHL

      Group size: 2

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      Group Info: Retrospective analysis of a longitudinal cohort of 21 VHL germline mutation carriers and retinal haemangioblastomas. All patients were categorized in two genotypic categories: missense mutations and truncating mutations. From this family, 2 individuals were suspected of VHL disease, but none developed RH.

      GroupPresentingHPOs: N/A

      GroupHPOFreeText: N/A

      GroupNotHPOs: HP:0009711 (retinal capillary hemangioma)

      GroupNotHPOFreeText: None of the VHL patients presented with retinal capillary hemangioma.

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Table S1

      Variant: p.R167Q (c.500G>A)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 2216, https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: N/A

      gnomAD: 0.000003977, https://gnomad.broadinstitute.org/variant/3-10191507-G-A

      VariantEvidence: Chr3(GRCh37): g.10191507G>A

      MutationType: missense_variant; transition

      CivicName: R167Q (c.500G>A)

      MultipleGeneVariants: N/A

    2. 13 Missense p.(Leu89Pro) 0 1

      GroupID: 13

      DiseaseAssertion: VHL

      Group size: 1

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      Group Info: Retrospective analysis of a longitudinal cohort of 21 VHL germline mutation carriers and retinal haemangioblastomas. All patients were categorized in two genotypic categories: missense mutations and truncating mutations. From this family, 1 individual was suspected of VHL disease, but did not develop RH.

      GroupPresentingHPOs: N/A

      GroupHPOFreeText: N/A

      GroupNotHPOs: HP:0009711 (retinal capillary hemangioma)

      GroupNotHPOFreeText: This VHL patient did not presented with retinal capillary hemangioma.

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Table S1

      Variant: p.L89P (c.266T>C)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 182979, https://www.ncbi.nlm.nih.gov/clinvar/variation/182979/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: Chr3(GRCh37): g.10183797T>C

      MutationType: missense_variant; transition

      CivicName: L89P (c.266T>C)

      MultipleGeneVariants: N/A

    3. 9 Frameshift p.(Lys159Glufs*15) 5 3

      GroupID: 9

      DiseaseAssertion: VHL

      Group size: 8

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      Group Info: Retrospective analysis of a longitudinal cohort of 21 VHL germline mutation carriers and retinal haemangioblastomas. All patients were categorized in two genotypic categories: missense mutations and truncating mutations. From this family, eight individuals were suspected of VHL disease. 5/8 individuals developed RH, while 3/8 did not.

      GroupPresentingHPOs: HP:0009711 (retinal capillary hemangioma)

      GroupHPOFreeText: Five patients presented with retinal capillary hemangioma.

      GroupNotHPOs: N/A

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Table S1

      Variant: p.L159Es*15 (c.473dup)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: N/A

      CAID: CA432423097, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.473dup

      gnomAD: N/A

      VariantEvidence: Chr3(GRCh37): g.10191480dup

      MutationType: duplication; plus_1_frameshift_variant

      CivicName: L159Es*15 (c.473dup)

      MultipleGeneVariants: N/A

    1. GroupID: 4485

      DiseaseAssertion: assumed VHL

      GroupSize: 51

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: Familial VHL stated; 51 family members recorded with this mutation.

      GroupPresentingHPOs: HP:0010797; HP:0005584; HP:0009711; HP:0002666 (hemangioblastoma, renal cell carcinoma, retinal capillary hemangioma, pheochromocytoma)

      GroupHPOFreeText: 25 of the 51 family members had an angioma (retinal hemangioma), 29 had RCC, 32 members had CNS hemangioblastoma, and 1 had a pheochromocytoma.

      GroupNotHPOs: N/A

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: 8956040 (confirmed Zbar 1996 type 2 family 4485NCI)

      SupplementalData: N/A

      Variant: p.L158P (c.473T>C)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: p.L158P (c.686T>C)

      ClinVarID: 182980 https://www.ncbi.nlm.nih.gov/clinvar/variation/182980/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transition

      CivicName: L158P (c.473T>C)

      MultipleGeneVariants: N/A

    2. GroupID: 3177

      DiseaseAssertion: assumed VHL

      GroupSize: 6

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: Familial VHL stated; 6 family members recorded with this mutation.

      GroupPresentingHPOs: HP:0010797; HP:0009711 (hemangioblastoma, retinal capillary hemangioma)

      GroupHPOFreeText: 4/6 members had a retinal angioma (hemangioma), and 3/6 members had CNS hemangioblastoma

      GroupNotHPOs: HP:0002666; HP:0005584 (pheochromocytoma, renal cell carcinoma)

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R82_V84del (c.244_252delCGCGTCGTG)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: p.153delR-V-V (c.457delCGCGTCGTC)

      ClinVarID: N/A

      CAID: CA658820713 https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.244_252delCGCGTCGTG

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: inframe_deletion

      CivicName: R82_V84del (c.244_252del)

      MultipleGeneVariants: N/A

    3. PatientID: None

      KindredID: 4401

      Case: Sex, Age, Ethnicity Unknown

      DiseaseAssertion: assumed VHL

      FamilyInfo: Familial VHL stated; although only 1 family member recorded.

      CasePresentingHPOs: HP:0010797 (hemangioblastoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0002666; HP:0009711; HP:0005584 (Pheochromocytoma, retinal capillary hemangioma, renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.S111R (c.333C>G)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: p.S182R (c.546C>G)

      ClinVarID: 223187 https://www.ncbi.nlm.nih.gov/clinvar/variation/223187/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: S111R (c.333C>G)

      MultipleGeneVariants: N/A

    4. GroupID: 2693

      DiseaseAssertion: assumed VHL

      GroupSize: 7

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: Familial VHL stated; 7 family members recorded with this mutation.

      GroupPresentingHPOs: HP:0010797; HP:0009711; HP:0005584 (hemangioblastoma, retinal capillary hemangioma, renal cell carcinoma)

      GroupHPOFreeText: 3/7 members had a retinal angioma (hemangioma), 3 had RCC, and 3 members had CNS hemangioblastoma

      GroupNotHPOs: HP:0002666 (pheochromocytoma)

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: Possibly previously published in PMID 9829911 [F76del (c.226_228del) family (ID 183)]; as well as one member in a future publication: PMID: 32179488

      SupplementalData: N/A

      Variant: p.F76del (c.226_228delTTC)

      CaseProblemVariantFreeText: NM_000551.3:c.226_228delTTC automaps to NM_000551.3:c.227_229delTCT due to redundancy

      LegacyVariant: p.147delF (c.439delTTC)

      ClinVarID: N/A

      CAID: CA357012 https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.226_228delTTC

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: inframe_deletion

      CivicName: F76del (c.224_226delTCT)

      MultipleGeneVariants: N/A

    5. PatientID: None

      KindredID: 3630

      Case: Sex, Age, Ethnicity Unknown

      DiseaseAssertion: assumed VHL

      FamilyInfo: Familial VHL stated; although only 1 family member recorded.

      CasePresentingHPOs: HP:0010797; HP:0009711 (hemangioblastoma; retinal capillary hemangioma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0002666; HP:0005584 (Pheochromocytoma, renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.S80I (c.239G>T)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: p.S151I (c.452G>T)

      ClinVarID: 625229 https://www.ncbi.nlm.nih.gov/clinvar/variation/625229/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: S80I (c.239G>T)

      MultipleGeneVariants: N/A

    1. III:8)

      PatientID: III:8

      KindredID: 1

      Case: M, Age Unknown, Chinese

      DiseaseAssertion: Asymptomatic

      FamilyInfo: One of the proband's maternal cousins was a healthy individual that was demonstrated to carry the same mutation, as revealed by Sanger sequencing: III:8, female. A second cousin, a 28 year old male (the proband III:2), was also found to have the same mutation and had a hemangioblastoma. A further five affected members of the family had previously died from VHL syndrome, including III:8's father (II:5), paternal grandfather (I:1), an uncle (II:3) and aunt (II:2) and a sister (III:10). These deceased family members had not previously been tested for mutations in the VHL gene. Pedigree: see Figure 1.

      CasePresentingHPOs: None

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: All members of the family were examined by two experienced surgeons. Diagnoses of VHL syndrome were determined via investigation of medical histories and performance of physical examinations, including a complete neurological examination and brain magnetic resonance imaging (MRI)

      CasePreviousTesting: N/A

      PreviouslyPublished: NA

      SupplementalData: NA

      Variant: p.H115Q (c.345C>A)

      ClinVar: 496057 https://www.ncbi.nlm.nih.gov/clinvar/variation/496057/

      CAID:N/A

      gnomAD: N/A

      VariantEvidence: RNA extracted from peripheral blood samples obtained from patients in the pedigree and the healthy controls. No significant difference in the expression of pVHL was demonstrated between the mutated and wild type cDNA, as demonstrated by RT-sqPCR analysis.

      MutationType: missense_variant;transversion

      CivicName: H115Q (c.345C>A)

      MultipleGeneVariants: N/A

    2. III:4

      PatientID: III:4

      KindredID: 1

      Case: M, Age Unknown, Chinese

      DiseaseAssertion: Asymptomatic

      FamilyInfo: One of the proband's maternal cousins was a healthy individual that was demonstrated to carry the same mutation, as revealed by Sanger sequencing: III:8, female. A second cousin, a 28 year old male (the proband III:2), was also found to have the same mutation and had a hemangioblastoma. A further five affected members of the family had previously died from VHL syndrome, including pateint III:4's father (II:3), paternal grandfather (I:1), an uncle (II:5) and aunt (II:2) and a cousin (III:10). These deceased family members had not previously been tested for mutations in the VHL gene. Pedigree: see Figure 1.

      CasePresentingHPOs: None

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: All members of the family were examined by two experienced surgeons. Diagnoses of VHL syndrome were determined via investigation of medical histories and performance of physical examinations, including a complete neurological examination and brain magnetic resonance imaging (MRI)

      CasePreviousTesting: N/A

      PreviouslyPublished: NA

      SupplementalData: NA

      Variant: p.H115Q (c.345C>A)

      ClinVar: 496057 https://www.ncbi.nlm.nih.gov/clinvar/variation/496057/

      CAID:N/A

      gnomAD: N/A

      VariantEvidence: RNA extracted from peripheral blood samples obtained from patients in the pedigree and the healthy controls. No significant difference in the expression of pVHL was demonstrated between the mutated and wild type cDNA, as demonstrated by RT-sqPCR analysis.

      MutationType: missense_variant;transversion

      CivicName: H115Q (c.345C>A)

      MultipleGeneVariants: N/A

    1. CP 598C>T mutation had been inherited from the mother

      PatientID: N/A

      KindredID: 8

      Case: F, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: Asymptomatic

      FamilyInfo: Her child had polycythemia and had two VHL gene mutations; one he obtained from his father and another he obtained from this patient, the mother. The entire family had normal sensitivity of erythroid progenitors to Epo.

      CasePresentingHPOs: N/A

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R200W (c.598C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2232 https://www.ncbi.nlm.nih.gov/clinvar/variation/2232/

      CAID: CA020510 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.598C%3ET

      gnomAD: 0.0002016 https://gnomad.broadinstitute.org/variant/3-10191605-C-T

      VariantEvidence: The patient is heterozygous for the 598C>T mutation.

      MutationType: missense_variant;transition

      CivicName: R200W (c.598C>T)

      MultipleGeneVariants: N/A

    1. 5 nonsenses

      GroupID/ KindredID: 12

      DiseaseAssertion: assumed VHL

      GroupSize: 3

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: Chinese

      GroupAgeRange: N/A

      GroupInfo: With a family history, 3 family members were recorded in this group with the given mutation.

      GroupPresentingHPOs: HP:0010797; HP:0009711; HP:0005584; HP:0001732 (hemangioblastoma; retinal capillary hemangioma; renal cell carcinoma; pancreatic lesion)

      GroupHPOFreeText: 2 members had CNS hemangioblastomas, 1 has retinal angiomas, 1 had RCC, and 1 had a pancreatic abnormality

      GroupNotHPOs: HP:0002666 (pheochromocytoma)

      GroupNotHPOFreeText: No family members appeared to have the above manifestation.

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.Q96* (c.286C>T)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: p.Q96* (c.499C>T)

      ClinVarID: 428804 https://www.ncbi.nlm.nih.gov/clinvar/variation/428804/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: stop_gained

      CivicName: Q96* (c.286C>T)

      MultipleGeneVariants: N/A

    1. P15F9F10850yyOU20/250nn20/25nnn

      PatientID: 15

      KindredID: 9

      Case: F, 59Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Another individual (patient 16) from the same family was reported but their relationship to patient 15 was not indicated. This individual had VHL disease but the types of tumours they had were not specified.

      CasePresentingHPOs: HP:0010797 (hemangioblastoma)

      CaseHPOFreeText: Patient has VHL disease with CNS hemangioblstoma. She also had other tumours, but specific types of tumours were not indicated.

      CaseNotHPOs: HP:0009711 (Retinal capillary hemangioma)

      CaseNotHPOFreeText: Patient did not have CNS hemangioblastoma or Retinal capillary hemangiomas.

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.S65W (c.194C>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 43597, https://www.ncbi.nlm.nih.gov/clinvar/variation/43597/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: S65W (c.194C>G)

      MultipleGeneVariants: N/A

    2. P7F3M67nnOU20/200nn20/20nnn

      PatientID: 7

      KindredID: 3

      Case: M, 7Y0M, Ethnicity Unknown

      DiseaseAssertion: Asymptomatic

      FamilyInfo: A family pedigree is provided in figure 1. Both the father and sister of patient 7 were found to have the same mutation. His father had retinal, cerebellar and spinal hemangioblastomas, as well as retinal detachment. His 10-year-old sister did not have any known tumours.

      CasePresentingHPOs: N/A

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0009711 (Retinal capillary hemangioma)

      CaseNotHPOFreeText: Patient did not have retinal capillary hemangioma or other known tumours.

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R161* (c.481C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2217, https://www.ncbi.nlm.nih.gov/clinvar/variation/2217/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: stop_gained

      CivicName: R161* (c.481C>T)

      MultipleGeneVariants: N/A

    3. P4F2F1266nnOU20/200nn20/20nnn

      PatientID: 4

      KindredID: 2

      Case: F, 16Y0M, Ethnicity Unknown

      DiseaseAssertion: Asymptomatic

      FamilyInfo: A family pedigree is provided. Patient’s mother and grandmother have the same mutation. Patient’s mother had a retinal capillary hemangioma and lamellar macular hole in the left eye. The grandmother did not have retinal capillary hemangioma, but she had other tumours that were not mentioned.

      CasePresentingHPOs: N/A

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0001737 (Retinal capillary hemangioma)

      CaseNotHPOFreeText: Patient did not have retinal capillary hemangiomas.

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.P192L (c.575C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 526677, https://www.ncbi.nlm.nih.gov/clinvar/variation/526677/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transition

      CivicName: P192L (c.575C>T)

      MultipleGeneVariants: N/A

    1. patients 1

      PatientID: 1

      KindredID: 1

      Case: Gender Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: Renal cell carcinoma

      FamilyInfo: The paper stated that the patient had family members with the same mutation.

      CasePresentingHPOs: HP:0005584 (Renal cell carcinoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.V170D(c.509T>A)

      LegacyVariant: p.V170D(c.722T>A)

      ClinVarID: 625256, http://www.ncbi.nlm.nih.gov/clinvar/variation/625256

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: V170D(c.509T>A)

      MultipleGeneVariants: N/A

    1.  c.509T>A15 (18)2a/b45 (16–71)HBr, HBc, HBsc, RCC, pNET, Pheo, Cr, Cp

      GroupID: None

      DiseaseAssertion: VHL

      GroupSize: 15 GroupNumberMales: N/A GroupNumberFemales: N/A GroupEthnicity: Dutch GroupAgeRange: 16-71

      GroupInfo: 15 out of 82 individuals were grouped together based on all having the c.509T>A of the VHL gene. This individual was classified as 2a/b meaning that it included de novo VHL mutation carriers or VHL mutation carriers having a family history with diagnosis of pheochromocytoma and other VHL related manifestations.

      GroupPresentingHPOs: HP:0009711; HP:0006880; HP:0009713; HP:0005584; HP:0030405; HP:0002666; HP:0000107; HP:0001737 (Retinal capillary hemangioma, Cerebellar hemangioblastoma, Spinal hemangioblastoma, Renal cell carcinoma, Pancreatic endocrine tumor, Pheochromocytoma, Renal cyst, Pancreatic cysts)

      GroupHPOFreeText: N/A

      GroupNotHPOs: N/A

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: 31087189

      SupplementalData: N/A

      Variant: p.V170D (c.509T>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 625256, https://www.ncbi.nlm.nih.gov/clinvar/variation/625256/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: V170D (c.509T>A)

      MultipleGeneVariants: N/A

    1. PatientID: Carrier 3 (son to proband 2)

      KindredID: 1

      Case: M, Age Unknown, Chinese

      DiseaseAssertion: Asymptomatic

      FamilyInfo: The family with PCCs includes 56 members of 4 generations, and is from Gansu Province. Among them, 7 extended over 3 generations were diagnosed as PCCs. One died before admission and another died due to a cardiovascular incident. Five were initially evaluated; evaluation included medical history, physical examination, abdominal ultrasonography, CT or MRI and laboratory tests. All 5 probands of this family underwent surgical resection. The tumors were benign under pathological examinations. There were no recurrence and malignant transformation over the period of up to 23-year follow-up. Twenty members, including 5 live patients and 15 direct relatives, accepted genetic examination.

      CasePresentingHPOs: N/A

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.H125P (c.374A>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223201, https://www.ncbi.nlm.nih.gov/clinvar/variation/223201/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: H125P (c.374A>C)

      MultipleGeneVariants: N/A

    1. 2

      PatientID: Unknown

      KindredID: 2

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: 5 members of this family were found to have the mutation and pheochromocytoma. Other declared diseases in this family are: paraganglioma, hemangioblastoma, lung metastases and spinal hemangioblastoma but the presenting patient is unclear.Relationship between these family members is unclear.

      CohortInfo: Patients with the diagnosis of familial or sporadic PHEO and their relatives.

      CasePresentingHPOs: HP:0002666 (Pheochromocytoma)

      CaseHPOFreeText: Follow-up 18 years later but it is unclear if this pertained to all affected family members.

      CaseNotHPOs: HP:0005584 (Renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Table 1

      Variant: p.P138= (c.414A>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223206, https://www.ncbi.nlm.nih.gov/clinvar/variation/223206/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: P138= (c.414A>G)

      MultipleGeneVariants: N/A

    2. nilateral PHEOs (Fig. 1a): her father (II:1) at age 73

      PatientID: II:1

      KindredID: 1

      Case: M, 73Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Look to proband (III:3) for extensive family info.

      CohortInfo: Patients with the diagnosis of familial or sporadic PHEO and their relatives.

      CasePresentingHPOs: HP:0002666; HP:0009713 (Pheochromocytoma, Spinal hemangioblastoma)

      CaseHPOFreeText: Deceased. Multiple spinal cord hemangioblastomas diagnosed after adrenal tumor resection. Unilateral pheochromocytoma diagnosed at age 73. Diagnosed with VHL type 2A.

      CaseNotHPOs: HP:0005584 (Renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Figure 1

      Variant: p.P138= (c.414A>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223206, https://www.ncbi.nlm.nih.gov/clinvar/variation/223206/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: P138= (c.414A>G)

      MultipleGeneVariants: N/A

    1. A 34-year-old pregnant woman with bilateral kidney tumors 9.5 and 2.5 cm in maximum diameter is presented. The larger tumor was clear renal cell carcinoma. The smaller contralateral tumor was focally HMB45 positive and had unusual histomorphology, including features resembling clear renal cell carcinoma with features of both t(6;11)- and t(X;17)/ASPL-TFE3 carcinomas. This tumor displayed a complex karyotype. A novel germ line mutation in the VHL gene (c.439A>G/p.I147V) was also identified in this patient.

      PatientID: 1 (case)

      KindredID: 1

      Case: F, 34Y0M, Ethnicity Unknown

      DiseaseAssertion: renal cell carcinoma

      FamilyInfo: No family information/ history was given. She was 19 weeks pregnant, however no other mentions of family.

      CasePresentingHPOs: HP:0006770 (clear cell renal cell carcinoma)

      CaseHPOFreeText: The patient had bilateral kidney tumors (9.5 and 2.5 cm in maximum diameter). The larger tumor was clear renal cell carcinoma. The smaller contralateral tumor was focally HMB45 positive and had unusual histomorphology, including features resembling clear renal cell carcinoma with features of both t(6;11)- and t(X;17)/ASPL-TFE3 carcinomas

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.I147V (c.439A>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 371833 https://www.ncbi.nlm.nih.gov/clinvar/variation/371833/

      CAID: N/A

      gnomAD: 0.00001061 https://gnomad.broadinstitute.org/variant/3-10188296-A-G

      VariantEvidence: HMB45-positive renal tumor resembling a t(6;11) carcinoma. There was a detection of translocation t(6;11) (Alpha/TFEB) and translocation t(X;17) (ASPL/TFE3). LOH was observed in the left-sides cRCC tumor.

      MutationType: missense_variant;transition

      CivicName: I147V (c.439A>G)

      MultipleGeneVariants: N/A

    1. 256C→T Ts Exon 1 86 CCC P P86S

      GroupID: 14

      DiseaseAssertion: VHL

      GroupSize: 3

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: French

      GroupAgeRange: N/A

      GroupInfo: 3 patients are listed with this mutation; any relation is unknown.

      GroupPresentingHPOs: HP:0009711 (retinal capillary hemangioma)

      GroupHPOFreeText: 1 member is said to have retinal HBs. Other manifestations are not clearly stated.

      GroupNotHPOs: HP:0009711 (retinal capillary hemangioma)

      GroupNotHPOFreeText: the other 2 members are specifically said to not have retinal HBs

      GroupPreviousTesting: N/A

      PreviouslyPublished: Possibly published in PMID:15300849

      SupplementalData: N/A

      Variant: p.P86S (c.256C>T)

      CaseProblemVariantFreeText: N.A

      LegacyVariant: N/A

      ClinVarID: 178692 https://www.ncbi.nlm.nih.gov/clinvar/variation/178692/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transition

      CivicName: P86S (c.256C>T)

      MultipleGeneVariants: N/A

    2. 226–228delTTC In Exon 1 76 TTC F In-frame deletion

      GroupID: 9

      DiseaseAssertion: VHL

      GroupSize: 2

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: French

      GroupAgeRange: N/A

      GroupInfo: 2 patients are listed with this mutation; any relation is unknown.

      GroupPresentingHPOs: N/A

      GroupHPOFreeText: Manifestations are not clearly stated.

      GroupNotHPOs: HP:0009711 (retinal capillary hemangioma)

      GroupNotHPOFreeText: both members are said to not have retinal HBs

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.F76del (c.226_228delTTC)

      CaseProblemVariantFreeText: N.A

      LegacyVariant: N/A

      ClinVarID: N/A

      CAID: CA357012 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.226_228delTTC

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: inframe_deletion

      CivicName: F76del (c.226_228delTTC)

      MultipleGeneVariants: N/A

    3. IVS1 + 1G→A Ts IVS1 Donor splice site — — Splice

      GroupID: 24

      Case: Age unknown, Sex unknown, French

      DiseaseAssertion: VHL

      FamilyInfo: None - one member documented here

      CasePresentingHPOs: HP:0009711 (retinal capillary hemangioma)

      CaseHPOFreeText: other manifestations are not clearly stated.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: Possibly published in PMID:15300849

      SupplementalData: N/A

      Variant: c.340+1G>A

      CaseProblemVariantFreeText: Written in paper as IVS1+1G>A

      LegacyVariant: N/A

      ClinVarID: 223190 https://www.ncbi.nlm.nih.gov/clinvar/variation/223190/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: splice_site_variant

      CivicName: Splice Site (c.340+1G>A)

      MultipleGeneVariants: N/A

    4. 466T→G Tv Exon 3 156 TAT Y Y156D

      GroupID: 37

      Case: Age unknown, Sex unknown, French

      DiseaseAssertion: VHL

      FamilyInfo: None - one member documented here

      CasePresentingHPOs: N/A

      CaseHPOFreeText: other manifestations are not clearly stated.

      CaseNotHPOs: HP:0009711 (retinal capillary hemangioma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: Possibly published in PMID:15300849

      SupplementalData: N/A

      Variant: p.Y156D (c.466T>G)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: N/A

      CAID: CA351756043 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.466T%3EG

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: Y156D (c.466T>G)

      MultipleGeneVariants: N/A

    5. 331A→T Tv Exon 1 111 AGC S S111C

      GroupID: 21

      DiseaseAssertion: VHL

      GroupSize: 3

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: French

      GroupAgeRange: N/A

      GroupInfo: 3 patients are listed with this mutation; any relation is unknown.

      GroupPresentingHPOs: HP:0009711 (retinal capillary hemangioma)

      GroupHPOFreeText: all 3 members are said to have retinal HBs. Other manifestations are not clearly stated.

      GroupNotHPOs: N/A

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.S111C (c.331A>T)

      CaseProblemVariantFreeText: N.A

      LegacyVariant: N/A

      ClinVarID: 565557 https://www.ncbi.nlm.nih.gov/clinvar/variation/565557/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: S111C (c.331A>T)

      MultipleGeneVariants: N/A

    1. PatientID: 6

      KindredID: 6

      Case: M, 11Y0M, Korean

      DiseaseAssertion: VHL

      FamilyInfo: No family history of VHL.

      CohortInfo: 26 Korean patients with more than one HAB in the CNS or eye, a single HAB in the CNS or retina plus a visceral manifestation (multiple renal, pancreatic or hepatic cysts, pheochromocytoma and RCC) and positive family history plus any one of the above manifestations.

      CasePresentingHPOs: HP:0009711; HP:0005562 (Retinal capillary hemangioma, Multiple renal cysts)

      CaseHPOFreeText: Multiple or bilateral retinal hemangioblastomas. Diagnosed with VHL Type 1.

      CaseNotHPOs: HP:0002666; HP:0005584; HP:0010797 (Pheochromocytoma, Renal cell carcinoma, Hemangioblastoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.E70K (c.208G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 43598, https://www.ncbi.nlm.nih.gov/clinvar/variation/43598/

      CAID: N/A

      gnomAD: 0.000004365, https://gnomad.broadinstitute.org/variant/3-10183739-G-A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: E70K (c.208G>A)

      MultipleGeneVariants: N/A

    2. PatientID: 39

      KindredID: 39

      Case: F, 24Y0M, Korean

      DiseaseAssertion: VHL

      FamilyInfo: Family negative for VHL.

      CohortInfo: 26 Korean patients with more than one HAB in the CNS or eye, a single HAB in the CNS or retina plus a visceral manifestation (multiple renal, pancreatic or hepatic cysts, pheochromocytoma and RCC) and positive family history plus any one of the above manifestations.

      CasePresentingHPOs: HP:0009711; HP:0010797; HP:0005584 (Retinal capillary hemangioma, Hemangioblastoma, Renal cell carcinoma)

      CaseHPOFreeText: CNS and retinal hemangioblastomas. Multiple or bilateral renal cell carcinoma. Diagnosed with VHL Type 1. Phenotypes revised from previous reports (see “PreviouslyPublished” section below for PMID).

      CaseNotHPOs: HP:0002666 (Pheochromocytoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 19270817

      SupplementalData: N/A

      Variant: p.C162R (c.484T>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 496067, https://www.ncbi.nlm.nih.gov/clinvar/variation/496067/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: C162R (c.484T>C)

      MultipleGeneVariants: N/A

    3. PatientID: 46

      KindredID: 46

      Case: M, 48Y0M, Korean

      DiseaseAssertion: VHL

      FamilyInfo: Family history negative for VHL.

      CohortInfo: 26 Korean patients with more than one HAB in the CNS or eye, a single HAB in the CNS or retina plus a visceral manifestation (multiple renal, pancreatic or hepatic cysts, pheochromocytoma and RCC) and positive family history plus any one of the above manifestations.

      CasePresentingHPOs: HP:0005584 (Renal cell carcinoma)

      CaseHPOFreeText: Multiple or bilateral renal cell carcinoma. Diagnosed with VHL Type 1.

      CaseNotHPOs: HP:0009711; HP:0002666; HP:0010797 (Retinal capillary hemangioma, Pheochromocytoma, Hemangioblastoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: “Shin, K. H., Park, K. J., Kim, S. W., Lee, S. H., Lee, S.-E., Jung, H.-W. et al. Germline mutations of VHL gene in Korean von Hippel–Lindau disease patients. J. Korean Cancer 28, 544–554 (1996).” (Reference 19 in current publication).

      SupplementalData: N/A

      Variant: p.R108Hfs*23 (c.323_324del)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: cDna and protein (i.e. c.536delGC deletion) do not match reference sequence. “This mutation, which reported in Shin et al., 1996 described as the reference 19 in our manuscript, was also corrected to c.323_324delGC, p.Arg108Hisfs*23 based on transcript NM_000551.3 in the human mutation database as below” (Author Rhee comment).

      ClinVarID: N/A

      CAID: CA916832609, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/by_canonicalid?canonicalid=CA916832609

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: deletion; minus_2_frameshift_variant

      CivicName: R108Hfs*23 (c.323_324del)

      MultipleGeneVariants: N/A

    1. F12c.414A>G/WTVHL p.Pro138Pro/WTNNNNVHL disease—

      PatientID: III (granddaughter)

      KindredID: F12

      Case: F, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: 4 members of this family (I.mother, II.daughter, II.son, III.granddaughter) presented with pheochromocytoma and were found to have the P138= (c.414A>G) germline variant of the VHL gene.

      CasePresentingHPOs: HP:0002666 (Pheochromocytoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.P138= (c.414A>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223206, https://www.ncbi.nlm.nih.gov/clinvar/variation/223206/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition; synonymous_variant

      CivicName: P138= (c.414A>G)

    2. F5

      PatientID: None (Mother)

      KindredID: F5

      Case: F, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: Asymptomatic

      FamilyInfo: N/A

      CasePresentingHPOs: N/A

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.G144R (c.430G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 576437, https://www.ncbi.nlm.nih.gov/clinvar/variation/576437/

      CAID: N/A

      gnomAD: 0.000003976, https://gnomad.broadinstitute.org/variant/3-10188287-G-A?dataset=gnomad_r2_1

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: G144R (c.430G>A) and c.340+694_340+711dup

      MultipleGeneVariants: N/A

    1. PatientID: III-7

      KindredID: 2

      Case: M, Age Unknown, German

      DiseaseAssertion: VHL (Asymptomatic)

      FamilyInfo: Family 2 is comprised of 131 individuals over six generations including 65 members (50% of those at risk) with VHL type 2A (72% clinically affected, 12% obligate carriers, and 15% positive genetic analysis alone) and nearly equal gender distribution for affected status (54% male). An additional six members (II-3, II-4, II -5, III-1, III-2, and III-3) are suspected to have VHL without confirmation. Of note, the nuclear family (branch IV-13) contains 13 siblings, of whom 12 have been diagnosed with pheo. The presenting tumor types in Family 2 were pheo (70%), HB (17%), and RA (13%). No Family 2 member had RCC as the initial manifestation. Distribution of HB and RA are not described except for patient IV-8.

      CasePresentingHPOs: N/A

      CaseHPOFreeText: Deceased.

      CaseNotHPOs: HP:0005584; HP:0002666 (Renal cell carcinoma, Pheochromocytoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 7759077

      SupplementalData: N/A

      Variant: p.Y98H (c.292T>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2223, https://www.ncbi.nlm.nih.gov/clinvar/variation/2223/

      CAID: N/A

      gnomAD: 0.00001343, https://gnomad.broadinstitute.org/variant/3-10183823-T-C

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: Y98H (c.292T>C)

      MultipleGeneVariants: N/A

    2. PatientID: IV-7

      KindredID: 2

      Case: M, Age Unknown, German

      DiseaseAssertion: VHL (Asymptomatic)

      FamilyInfo: Family 2 is comprised of 131 individuals over six generations including 65 members (50% of those at risk) with VHL type 2A (72% clinically affected, 12% obligate carriers, and 15% positive genetic analysis alone) and nearly equal gender distribution for affected status (54% male). An additional six members (II-3, II-4, II -5, III-1, III-2, and III-3) are suspected to have VHL without confirmation. Of note, the nuclear family (branch IV-13) contains 13 siblings, of whom 12 have been diagnosed with pheo. The presenting tumor types in Family 2 were pheo (70%), HB (17%), and RA (13%). No Family 2 member had RCC as the initial manifestation. Distribution of HB and RA are not described except for patient IV-8.

      CasePresentingHPOs: N/A

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0005584; HP:0002666 (Renal cell carcinoma, Pheochromocytoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 7759077

      SupplementalData: N/A

      Variant: p.Y98H (c.292T>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2223, https://www.ncbi.nlm.nih.gov/clinvar/variation/2223/

      CAID: N/A

      gnomAD: 0.00001343, https://gnomad.broadinstitute.org/variant/3-10183823-T-C

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: Y98H (c.292T>C)

      MultipleGeneVariants: N/A

    3. PatientID: V-18

      KindredID: 2

      Case: M, Age Unknown, German

      DiseaseAssertion: VHL

      FamilyInfo: Family 2 is comprised of 131 individuals over six generations including 65 members (50% of those at risk) with VHL type 2A (72% clinically affected, 12% obligate carriers, and 15% positive genetic analysis alone) and nearly equal gender distribution for affected status (54% male). An additional six members (II-3, II-4, II -5, III-1, III-2, and III-3) are suspected to have VHL without confirmation. Of note, the nuclear family (branch IV-13) contains 13 siblings, of whom 12 have been diagnosed with pheo. The presenting tumor types in Family 2 were pheo (70%), HB (17%), and RA (13%). No Family 2 member had RCC as the initial manifestation. Distribution of HB and RA are not described except for patient IV-8.

      CasePresentingHPOs: HP:0002666 (Pheochromocytoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0005584 (Renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 7759077

      SupplementalData: N/A

      Variant: p.Y98H (c.292T>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2223, https://www.ncbi.nlm.nih.gov/clinvar/variation/2223/

      CAID: N/A

      gnomAD: 0.00001343, https://gnomad.broadinstitute.org/variant/3-10183823-T-C

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: Y98H (c.292T>C)

      MultipleGeneVariants: N/A

    1. A 16-year-old male developed holocranial headache with projectile vomiting 3 years ago and subsequently developed sudden quadriparesis. Subsequent CECT revealed a posterior fossa fourth ventricle tumor with hydrocephalus. He underwent a suboccupalcraniatectomy with CI laminectomy with biopsy of the tumor along with VP shunt and then was referred to SGPGIMS. He underwent DSA embolization in view of hypervascular tumor and in preparation of excision. Subsequently, as his biopsy was suggestive of hemangioblastoma and he had a strong family history (his grandfather died of a brain tumor at the age of 45, his cousin brother was operated for a bilateral PCC and his paternal uncle was diagnosed with hemangioblastoma), he was suspected of VHL and CECT abdomen was done that revealed bilateral adrenal lesions and pancreatic and renal cysts; subsequently, elevated urinary MN/NMN (5,476 mcg/ml) confirmed bilateral PCC and he was taken up for bilateral adrenalectomy after adequate alpha blockade. HPE of adrenal lesion revealed bilateral tumors without hyperplasia. He was then transferred to neurosurgery unit where he underwent re-exploration and complete excision of brainstem hemangioblastoma. However, in postop period he gradually deteriorated and later developed septic shock and eventually succumbed.Genetic mutation revealed heterozygous VHL: C277G known pathogenic variant ({"type":"entrez-nucleotide","attrs":{"text":"NM_000551.3","term_id":"319655736","term_text":"NM_000551.3"}}NM_000551.3 [VHL]:c.277G>Cin exon 1. Protein change glycine to arginine [p. Gly93Arg]).

      PatientID: 3

      KindredID: 3

      Case: M, 16Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Strong family history. His grandfather died of a brain tumor at the age of 45, his cousin brother was operated for a bilateral PCC and his paternal uncle was diagnosed with hemangioblastoma.

      CasePresentingHPOs: HP:0006748, HP:0000107, HP:0001737, HP:0010797 (Adrenal pheochromocytoma, renal cyst, pancreatic cysts, hemangioblastoma)

      CaseHPOFreeText: Subsequent CECT revealed a posterior fossa fourth ventricle tumor with hydrocephalus. He underwent a suboccupalcraniatectomy with CI laminectomy with biopsy of the tumor along with VP shunt and then was referred to SGPGIMS. He underwent DSA embolization in view of hypervascular tumor and in preparation of excision. He had a hemangioblastoma of the brainstem.

      CaseNotHPOs: HP:0005584 (Renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.G93R (c.277G>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223174 https://www.ncbi.nlm.nih.gov/clinvar/variation/223174/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: The patient was heterozygous for the mutation.

      MutationType: missense_variant;transversion

      CivicName: G93R (c.277G>C)

      MultipleGeneVariants: N/A

    1. PatientID: 139 (Second cousin)

      KindredID: 18

      Case: F, 50Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: 11 members of this family tested positive for the c.712C>T/c.499C>T germline variant of the VHL gene. 14 members of this family were assumed to carry this variant due to family history/personal medical history. Members of this family range from asymptomatic to multi-organ involvement (pheochromocytoma, pancreatic and renal cysts/tumors and hemangioblastomas). The family was previously reported in PMID 3942913.

      CasePresentingHPOs: HP:0009711; HP:0002666; HP:0005584 (Retinal capillary hemangioma, Pheochromocytoma, Renal cell carcinoma)

      CaseHPOFreeText: This patient presented with retinal angioma (16Y). Age of diagnosis of pheochromocytoma and renal cell carcinoma is unknown.

      CaseNotHPOs: HP:0000077; HP:0001732; HP:0010797 (Abnormality of the kidney, Abnormality of the pancreas, Hemangioblastoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 3942913 (some members of this family are previously reported (phenotypes only) but unable to match patients across papers with confidence)

      SupplementalData: Supplementary Table 1.

      Variant: p.R167W (c.499C>T)

      LegacyVariant: c.712C>T

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2218, https://www.ncbi.nlm.nih.gov/clinvar/variation/2218/

      CAID: N/A

      gnomAD: 0.000007954, https://gnomad.broadinstitute.org/variant/3-10191506-C-T?dataset=gnomad_r2_1

      VariantEvidence: A point mutation (c.712C>T) was found. Being that this variant is out of bound with respect to the NM_000551.3 reference sequence, it was concluded that this variant is noted in the Latif et al. (1993) reference and was translated to c.499C>T (NM_000551.3).

      MutationType: missense_variant;transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    2. PatientID: 25 (Second cousin)

      KindredID: 17

      Case: F, 9Y0M, Ethnicity Unknown

      DiseaseAssertion: Asymptomatic

      FamilyInfo: 6 members of this family tested positive for the c.496_506del germline variant of the VHL gene. ID 4 presented with CNS hemangioblastoma, retinal angioma and renal and pancreatic abnormalities. ID 1 and 8 were asymptomatic.ID 22 presented with CNS hemangioblastoma. ID 5 presented with retinal angioma. 2 members were assumed to have the variant but were not genetically confirmed (ID 20 and ID 11). ID 11 and 20 presented with CNS hemangioblastoma.

      CasePresentingHPOs: N/A

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0005584; HP:0002666; HP:0010797; HP:0000077; HP:0001732; HP:0009711 (Renal cell carcinoma, Pheochromocytoma, Hemangioblastoma, Abnormality of the kidney, Abnormality of the pancreas, Retinal capillary hemangioma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Supplementary Table 1.

      Variant: p.V166Sfs*4 (c.496_506del)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223229, https://www.ncbi.nlm.nih.gov/clinvar/variation/223229/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: It is unclear from the publication if this variant is reported according to legacy (AF010238.1) or NM_000551.3 reference sequence. Clinical notes from the case confirm the reference sequence to be NM_000551.3.

      MutationType: deletion; minus_2_frameshift_variant

      CivicName: V166SFS*4 (c.496_506del)

      MultipleGeneVariants: N/A

    1. In this study, we analyzed the correlations between frequent mutations in the VHL gene and clinical phenotypes in the largest Chinese VHL cohort to date. In total, we screened 540 patients from 187 unrelated VHL families and identified 126 different VHL mutations. Furthermore, we identified 19 frequent mutations and four mutation hotspots and further investigated the genotype–phenotype correlations. Notably, patients or families with the VHL disease have a range of different phenotypes. A variety of factors may contribute to this diversity of phenotypes, including the type of VHL mutation, the site of the mutation, and ethnic background.

      GroupID: 13

      DiseaseAssertion: assumed VHL

      GroupSize: 7

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: Chinese

      GroupAgeRange: 39.0Y +/- 13.1Y

      GroupInfo: Clinical characteristics of 258 patients from 80 unrelated Chinese families were reported in this study. Grouped by the 21 mutations found, 2 families with 7 members total were described, and phenotypic information was given for this group.

      GroupPresentingHPOs: HP:0010797; HP:0009711; HP:0005584; HP:0001737 (hemangioblastoma, retinal capillary hemangioma, renal cell carcinoma, pancreatic cysts)

      GroupHPOFreeText: 5 of the 7 members presented with CNS hemangioblastoma (with a mean age of 34.2Y), retinal capillary hemangioma in 1 member at 26Y, renal cell carcinoma in 3 members (at the mean age of 36.7Y), pancreatic cysts in 2 members (at the ages of 32 and 38Y)

      GroupNotHPOs: HP:0002666; HP:0009715 (pheochromocytoma, papillary cystadenoma of the epididymis)

      GroupNotHPOFreeText: No patients presented with pheochromocytoma or a papillary cystadenoma of the epididymis genital system tumor (epididymis or broad ligament)

      GroupPreviousTesting: N/A

      PreviouslyPublished: 29330336; 28388566

      SupplementalData: N/A

      Variant: p.I151S (c.452T>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223215 https://www.ncbi.nlm.nih.gov/clinvar/variation/223215/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: I151S (c.452T>G)

      MultipleGeneVariants: N/A

    2. In our study, group 5 represented the c.262T > C p.Trp88Arg mutation that included 21 patients from 2 families (Figure 4 and Table 3). Variability was observed in the mean age at onset for CHB and RCC, which were 41.1 ± 9.1 (range = 29–62) and 37.4 ± 12.9 (range = 23–65), respectively.

      PatientID: 4

      KindredID: 1 (group ID 5)

      Case: F, 66Y0M, Chinese

      DiseaseAssertion: assumed VHL

      CohortInfo: Clinical characteristics of 258 patients from 80 unrelated Chinese families were reported in this study. Grouped by the 21 mutations found, 2 families with 21 members total were described, and phenotypic information was given for this group.

      FamilyInfo: In family 1, 17 members were described. The first generation describes 5 members with the VHL mutation, each passing on the gene to further generations. Patient 4’s two sons were passed this familial mutation. See Fig 4 for family pedigree; Table 3 for details.

      CasePresentingHPOs: HP:0010797 (hemangioblastoma)

      CaseHPOFreeText: She had CNS hemangioblastomas at 37Y

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.W88R (c.262T>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 496053 https://www.ncbi.nlm.nih.gov/clinvar/variation/496053/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: W88R (c.262T>C)

      MultipleGeneVariants: N/A

    3. In our study, group 5 represented the c.262T > C p.Trp88Arg mutation that included 21 patients from 2 families (Figure 4 and Table 3). Variability was observed in the mean age at onset for CHB and RCC, which were 41.1 ± 9.1 (range = 29–62) and 37.4 ± 12.9 (range = 23–65), respectively.

      PatientID: 2

      KindredID: 1 (group ID 5)

      Case: M, 58Y0M (deceased), Chinese

      DiseaseAssertion: asymptomatic

      CohortInfo: Clinical characteristics of 258 patients from 80 unrelated Chinese families were reported in this study. Grouped by the 21 mutations found, 2 families with 21 members total were described, and phenotypic information was given for this group.

      FamilyInfo: In family 1, 17 members were described. The first generation describes 5 members with the VHL mutation, each passing on the gene to further generations. Patient 2 had a daughter and son with this mutation. See Fig 4 for family pedigree; Table 3 for details.

      CasePresentingHPOs: N/A

      CaseHPOFreeText: Phenotypes for this patient were not described

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.W88R (c.262T>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 496053 https://www.ncbi.nlm.nih.gov/clinvar/variation/496053/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: W88R (c.262T>C)

      MultipleGeneVariants: N/A

    1. PatientID: N/A

      KindredID: 19

      Case: Sex, Age, Ethnicity Unknown

      DiseaseAssertion: pheochromocytoma

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0002666 (pheochromocytoma)

      CaseHPOFreeText: Nonsyndromic pheochromocytoma.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: RET, SDHB, SDHD

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167Q (c.500G>A)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: p.R167Q (c.713G>A)

      ClinVarID: 2216 https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: CA020454 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.500G%3EA

      gnomAD: 0.000003977 https://gnomad.broadinstitute.org/variant/3-10191507-G-A

      VariantEvidence: N/A

      MutationType: missense_variant; transition

      CivicName: R167Q (c.500G>A)

      MultipleGeneVariants: N/A

    2. GroupID/ KindredID: 14

      DiseaseAssertion: pheochromocytoma

      GroupSize: 2

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: 2 patients are listed with this mutation; any relation is unknown.

      GroupPresentingHPOs: HP:0002666 (pheochromocytoma)

      GroupHPOFreeText: 2 members are stated to have pheochromocytoma. Other manifestations are possible but not clear.

      GroupNotHPOs: N/A

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: RET, SDHB, SDHD

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.Y156C (c.467A>G)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: p.Y156C (c.680A>G)

      ClinVarID: 43600 https://www.ncbi.nlm.nih.gov/clinvar/variation/43600/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: Y156C (c.467A>G)

      MultipleGeneVariants: N/A

    3. PatientID: N/A

      KindredID: 7

      Case: Sex, Age, Ethnicity Unknown

      DiseaseAssertion: pheochromocytoma

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0002666 (pheochromocytoma)

      CaseHPOFreeText: Nonsyndromic pheochromocytoma.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: RET, SDHB, SDHD

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.E94* (c.280G>T)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: p.E94* (c.493G>T)

      ClinVarID: 584477 https://www.ncbi.nlm.nih.gov/clinvar/variation/584477/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: stop_gained

      CivicName: E94* (c.280G>T)

      MultipleGeneVariants: N/A

    1. Screening of the 27-yr-old son revealed asymptomatic retinal and spinal hemangioblastomas.

      PatientID: N/A (son 2)

      KindredID: 5

      Case: M, 27Y0M, Ethnicity Unknown

      DiseaseAssertion: assumed VHL

      FamilyInfo: His mother was confirmed with the mutation, and various manifestations of VHL. His two brothers also carried the mutation, one with adrenal pheochromocytomas as well. His grandmother and great uncle passed away likely due to VHL complications.

      CasePresentingHPOs: HP:0009711; HP:0009713 (retinal capillary hemangioma, spinal hemangioblastoma)

      CaseHPOFreeText: At 27Y screening showed an asymptomatic retinal and spinal hemangioblastoma

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R107H (c.320G>A)

      LegacyVariant: p.R107H

      CaseProblemVariantFreeText: As the publication only provides the p., the mutation was deduced to p.R107H (c.320G>A)

      ClinVarID: 223184 https://www.ncbi.nlm.nih.gov/clinvar/variation/223184/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R107H (c.320G>A)

      MultipleGeneVariants: N/A

    1. 26F/18FSB/L PCC+sPGLCNS haemangioblastomas, PNET, pancreatic cysts.Family history +VHL3c.499C>Tp.(Arg167Trp)Substitution (missense)Disease causing

      PatientID: 26

      KindredID: 26

      Case: F, 18Y0M, Asian Indian

      DiseaseAssertion: Assumed VHL

      FamilyInfo: Positive family history

      CasePresentingHPOs: HP:0002666; HP:0002668; HP:0010797; HP:0030405; HP:0001737 (pheochromocytoma; paraganglioma; hemangioblastomas; pancreatic endocrine tumor; pancreatic cysts)

      CaseHPOFreeText: patient had bilateral pheochromocytoma and sympathetic paraganglioma; CNS hemangioblastomas, PNETs and pancreatic cysts

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: RET/ SDHB/ SDHD/ NF1

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167W (c.499C>T)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 2218 https://www.ncbi.nlm.nih.gov/clinvar/variation/2218/

      CAID: N/A

      gnomAD: 0.000007954 https://gnomad.broadinstitute.org/variant/3-10191506-C-T

      VariantEvidence: N/A

      MutationType: missense_variant; transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    1. a 4-year-old male with bilateral adrenal pheochromocytomas

      PatientID: None

      KindredID: 1

      Case: M, 5Y0M, Ethnicity Unknown

      DiseaseAssertion: Adrenal pheochromocytoma

      FamilyInfo: The authors of the paper asserted that the mutation was de novo; the parents and sibling were tested and were not found to have the mutation.

      CasePresentingHPOs: HP:0006748; HP:0100553 (Adrenal pheochromocytoma, hemihypertrophy of the lower limb)

      CaseHPOFreeText: The 4Y male patient presented with excessive sweating, poor weight gain for 6 months, and left-sided hemihypertrophy affecting lower limbs. The patient was diagnosed with bilateral adrenal pheochromocytoma and underwent resection. A one year follow-up after bilateral resection found no recurrence.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R82L(c.245G>T)

      LegacyVariant: N/A

      ClinVarID: 526675, http://www.ncbi.nlm.nih.gov/clinvar/variation/526675

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: R82L(c.245G>T)

      MultipleGeneVariants: N/A

    1. II 0 10 No manifestations M c.194C > G (p.Ser65Trp)

      PatientID: II

      KindredID: 9

      Case: Sex Unknown, 10Y0M, Danish

      DiseaseAssertion: Asymptomatic

      FamilyInfo: Two other family members (9-I and 9-III) carried the same mutation. Patient I was asymptotic but patient III presented with two hemangioblastomas and two pancreatic cysts.

      CasePresentingHPOs: N/A

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 19863552, 25834951, 27966541, 27539272

      SupplementalData: N/A

      Variant: p.S65W(c.194C>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 43597 https://www.ncbi.nlm.nih.gov/clinvar/variation/43597/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName:S65W(c.194C>G)

      MultipleGeneVariants: N/A

    1. two cases

      PatientID: None

      KindredID: 2

      Case: M, 53Y0M, Ethnicity Unknown

      DiseaseAssertion: Assumed VHL

      FamilyInfo: One of the patient’s 3 children, a 25Y female, was found to carry the mutation but was asymptomatic.

      CasePresentingHPOs: HP:0006770; HP:0000107; HP:0001737 (Clear cell renal cell carcinoma, renal cysts, pancreatic cysts)

      CaseHPOFreeText: The patient presented with ccRCC, a renal cyst and a pancreatic nodule. The patient was asymptomatic except for mild hearing loss. The patient is deceased.

      CaseNotHPOs: HP:0010797; HP:0002666 (Hemangioblastoma, pheochromocytoma)

      CaseNotHPOFreeText: There was no evidence of hemangioblastomas of the brain or spine on MRI. Additionally, there was also no evidence of PCC on biochemical analysis, normal abdominal imaging results were present, and no retinal angiomas were found.

      CasePreviousTesting: SDHB, SDHC, SDHD

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: (c.-61_-51dup11)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: The mutation was a duplication in the VHL 5’ UTR involving the promoter.

      ClinVarID: 517152, http://www.ncbi.nlm.nih.gov/clinvar/variation/517152

      gnomAD: 0.0002825, https://gnomad.broadinstitute.org/variant/3-10141782-C-CTCCGCCCCGCG?dataset=gnomad_r3

      VariantEvidence: N/A

      MutationType: 5_prime_UTR_variant

      CivicName: c.-61_-51dup11

      MultipleGeneVariants: N/A

    1. Ninety-eight individuals were tested for VHL mutations--23 symptomatic and 75 asymptomatic individuals belonging to 16 families. Seven of the families had definite clinical criteria of VHL disease, five had sporadic hemangioblastoma, and four had CNS hemangioblastoma combined with minor visceral signs. Molecular genetic testing identified five germline mutations in six of the definite VHL families (sensitivity 85%), but none in the possible VHL and sporadic hemangioblastoma cases; four of these mutations had been previously described and one is a novel mutation present in two unrelated families. After patients carrying the mutation were identified, they underwent clinical screening and asymptomatic VHL-related lesions were identified in 43%.

      PatientID: 20

      KindredID: C

      Case: F, 13Y0M, Mexican (Latino/Admixed American)

      DiseaseAssertion: assumed VHL

      FamilyInfo: At least 13 members in this family presented with the mutation. 5 were asymptomatic (ages 9, 12, 13, 17, and 29Y). Other family manifestations include: cerebellar, spinal, retinal HBs, pancreatic, hepatic, suprarenal and kidney cysts, epididymal cystadenoma, and RCC. Fig 1 displays family pedigree.

      CasePresentingHPOs: HP:0006880 (cerebellar hemangioblastoma)

      CaseHPOFreeText: Patient had 1 cerebellar HB. She underwent surgery for this at the age of 9Y.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 20064270 (Here, the family cohort is given with a reportedly different variant of: p.G34Rfs*98 (c.99_100insA). Unclear of why this is or which most correctly corresponds to the family).

      SupplementalData: N/A

      Variant: p.T100Nfs*32 (c.298dupA)

      LegacyVariant: c.1012_1013insA

      CaseProblemVariantFreeText: The ‘1012_1013insA’ mutation is out-of-frame, leading to a modification of 31 amino acids downstream and a premature stop codon at nucleotides 5441-5443 (amino acid residue 131).

      ClinVarID: N/A

      CAID: CA2573129737 https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.298dupA

      gnomAD: N/A

      VariantEvidence: The modified amino acids are located in a domain common to all pVHL isoforms, the highly conserved cctbinding domain of the protein, involved in the formation of the pVHL elongin complex that is crucial for tumor suppression. Furthermore, the mutant protein is prematurely truncated and lacks the C-terminal half of exon 2 and exon 3 completely, which includes the nuclear export signal and the elongin C binding site of pVHL. Thus, this truncated protein would affect the two functional isoforms of pVHL: the major isoform pVHL30 and pVHL19, but not isoform 2, which lacks exon 2 but is not functional

      MutationType: plus_1_frameshift_variant; duplication

      CivicName: T100Nfs*32 (c.298dupA)

      MultipleGeneVariants: N/A

    1. Table 1)

      PatientID: N/A

      KindredID: 7

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: assumed VHL

      FamilyInfo: Family history not specified, but previously published paper (PMID: 20151405) states familial VHL. Patient’s variant reported as germline.

      CasePresentingHPOs: HP:0010797; HP:0009711; HP:0005584 (hemangioblastoma; retinal capillary hemangioma; renal cell carcinoma)

      CaseHPOFreeText: Patient had CNS HB, RA and a Grawitz tumor (RCC) (see table 1)

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 20151405

      SupplementalData:  N/A

      Variant: p.F76del (c.227_229del)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA357012, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.227_229delTCT

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: inframe_deletion

      CivicName: F76del (c.227_229del)

      MultipleGeneVariants: N/A

    2. Table 1)

      PatientID: N/A

      KindredID: 8

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Family history not specified, but previously published paper (PMID: 8641976) states that neither parent had the mutation, making it de novo. Patient’s variant reported as germline.

      CasePresentingHPOs: HP:0006880; HP:0011513 (Cerebellar hemangioblastoma, retinal angioma)

      CaseHPOFreeText: Patient had VHL disease; CNS-HB, RA (see table 1)

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 8641976

      SupplementalData:  N/A

      Variant: p.F76del (c.227_229del)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA357012, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.227_229delTCT

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: inframe_deletion

      CivicName: F76del (c.227_229del)

      MultipleGeneVariants: N/A

    3. Table 1)

      PatientID: N/A

      KindredID: 15

      Case: Sex Unknown, Age Unknown, Polish

      DiseaseAssertion: VHL

      FamilyInfo: Family history not mentioned. Patient’s variant reported as germline.

      CasePresentingHPOs: HP:0009711; HP:0010797 (retinal capillary hemangioma, hemangioblastoma)

      CaseHPOFreeText: Patient had VHL disease: CNS-HB and RHB (see table 1). Previously published paper (PMID: 12114495) indicates that patient is Polish.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 12114495

      SupplementalData: N/A

      Variant: p.F76del (c.227_229del)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA357012, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.227_229delTCT

      gnomAD: N/A

      VariantEvidence: Variant reported as c.226_228del and was mapped to c.227_229del.

      MutationType: inframe_deletion

      CivicName: F76del (c.227_229del)

      MultipleGeneVariants: N/A

    4. Table 1

      PatientID: N/A

      KindredID: 5

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: pheochromocytoma

      FamilyInfo: Transmission of the variant could not be determined since family history is not available. Patient’s variant reported as germline.

      CasePresentingHPOs: HP:0002666 (pheochromocytoma)

      CaseHPOFreeText: Patient had PH (see table 1). In 2011 from PMID:21463266, patient was reported to be 62Y0M.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 21463266

      SupplementalData:  N/A

      Variant: p.P59S (c.175C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 231233, https://www.ncbi.nlm.nih.gov/clinvar/variation/231233/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transition

      CivicName: P59S (c.175C>T)

      MultipleGeneVariants: N/A

    1. recognized in a large family that originated in Newfoundland but has some members who live in New Brunswick and Ontario. A collaborative investigation was begun in 1982 to document the number of affected members and the extent of their disease and to improve management of the disease. The condition has been documented in 38 members of the family, 28 living and 10 dead. The most common manifestations are retinal angioma (present in 60% of the gene carriers) and pheochromocytoma (present in 53%). Of the 28 living affected members 14 had been identified before the study began. Only 3 of the 14 patients in whom the disease was subsequently diagnosed presented with symptoms; in the remaining 11 the condition was detected by routine screening. Overall the mean age at the time of diagnosis was 23 years; in the 21 affected members of the fourth generation it was 18 years.

      PatientID: IV-36

      KindredID: 1

      Case: F, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: Assumed VHL

      FamilyInfo: 38 family members had been affected at the date of 1986, and 10 had passed away. Pheochromocytoma, cerebellar, spinal and retinal hemangioblastomas, and various cysts were clinical manifestations of VHL seen throughout the family. This patient (IV-36) appears to have inherited this disease from her mother, along with many of her siblings. See Fig. 1 for family pedigree.

      CasePresentingHPOs: HP:0009711 (retinal capillary hemangioma)

      CaseHPOFreeText: This member was examined in St. Johns at the Memorial University of Newfoundland. She had retinal angiomas which were not further described.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167W (c.499C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: Although never given at the time of this publication, the germline mutation is confirmed in this family in later publications (PMID 7987306; 29437867) for this specific family. It is only here assumed this member had the VHL mutation.

      ClinVarID: 2218, https://www.ncbi.nlm.nih.gov/clinvar/variation/2218/

      CAID: N/A

      gnomAD: 0.000007954, https://gnomad.broadinstitute.org/variant/3-10191506-C-T?dataset=gnomad_r2_1

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    2. recognized in a large family that originated in Newfoundland but has some members who live in New Brunswick and Ontario. A collaborative investigation was begun in 1982 to document the number of affected members and the extent of their disease and to improve management of the disease. The condition has been documented in 38 members of the family, 28 living and 10 dead. The most common manifestations are retinal angioma (present in 60% of the gene carriers) and pheochromocytoma (present in 53%). Of the 28 living affected members 14 had been identified before the study began. Only 3 of the 14 patients in whom the disease was subsequently diagnosed presented with symptoms; in the remaining 11 the condition was detected by routine screening. Overall the mean age at the time of diagnosis was 23 years; in the 21 affected members of the fourth generation it was 18 years.

      PatientID: IV-8

      KindredID: 1

      Case: F, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: Assumed VHL

      FamilyInfo: 38 family members had been affected at the date of 1986, and 10 had passed away. Pheochromocytoma, cerebellar, spinal and retinal hemangioblastomas, and various cysts were clinical manifestations of VHL seen throughout the family. This patient (IV-8) appears to have inherited this disease from her father, along with many of her siblings. See Fig. 1 for family pedigree.

      CasePresentingHPOs: HP:0006880; HP:0009711 (cerebellar hemangioblastoma, retinal capillary hemangioma)

      CaseHPOFreeText: This member was examined in St. Johns at the Memorial University of Newfoundland. She had cerebellar hemangioblastoma and retinal angiomas which were not further described.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167W (c.499C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: Although never given at the time of this publication, the germline mutation is confirmed in this family in later publications (PMID 7987306; 29437867) for this specific family. It is only here assumed this member had the VHL mutation.

      ClinVarID: 2218, https://www.ncbi.nlm.nih.gov/clinvar/variation/2218/

      CAID: N/A

      gnomAD: 0.000007954, https://gnomad.broadinstitute.org/variant/3-10191506-C-T?dataset=gnomad_r2_1

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    1. PatientID: 9

      KindredID: Unknown

      Case: M, 61Y0M, Dutch

      DiseaseAssertion: VHL

      FamilyInfo: Unknown - study sampled 15 patients from 5 families

      CasePresentingHPOs: HP:0001737; HP:0009711; HP:0006880; HP:0006706 (pancreatic cyst, retinal capillary hemangioma, cerebellar hemangioblastoma, Cystic liver disease)

      CaseHPOFreeText: pancreatic cyst, hemangioblastoma of the brain (cerebellar), retinal capillary hemangioma of the left eye, and liver cysts (cystic liver disease)

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.V170D (c.509T>A)

      LegacyVariant: p.V241D

      CaseProblemVariantFreeText: Variant determined through the assumption of the authors using Latif et al 1993 (AF010238.1) transcript and adding 71bp to the protein sequence.

      ClinVarID: 625256 https://www.ncbi.nlm.nih.gov/clinvar/variation/625256/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence:N/A

      MutationType: missense_variant;transversion

      CivicName: V170D (c.509T>A)

      MultipleGeneVariants: N/A

    1. missense variants

      PatientID: 35

      KindredID: 35

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0100634 (neuroendocrine neoplasm)

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0010797; HP:0006770; HP:0030405; HP:0009711; HP:0030421 (hemangioblastoma, clear cell renal cell carcinoma, pancreatic neuroendocrine neoplasm, retinal capillary hemangioma, Epididymal neoplasm)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 29437867 (Family 1 - see here for more info)

      SupplementalData: Yes; see table S1 for VHL subject genetic and manifestation information.

      Variant: p.S65A (c.193T>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223160 https://www.ncbi.nlm.nih.gov/clinvar/variation/223160

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: S65A (c.193T>G)

      MultipleGeneVariants: N/A

    2. 35 (45%) had missense variants,

      PatientID: 70

      KindredID: 70

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0010797; HP:0006770; HP:0009711 (hemangioblastoma; clear cell renal cell carcinoma; retinal capillary hemangioma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0100634; HP:0030405; HP:0030421 (Neuroendocrine neoplasm, pancreatic neuroendocrine neoplasm, Epididymal neoplasm)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Yes; see table S1 for VHL subject genetic and manifestation information.

      Variant: p.Q96P (c.287A>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 625233 https://www.ncbi.nlm.nih.gov/clinvar/variation/625233/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: Q96P (c.287A>C)

      MultipleGeneVariants: N/A

    3. 35 (45%) had missense variants

      PatientID: 8

      KindredID: 8

      Case: Age, Sex, Ethnicity unknown

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0010797; HP:0030405 (hemangioblastoma, pancreatic endocrine tumour)

      CaseHPOFreeText: patient was recorded with a CNS hemangioblastoma and a pancreatic endocrine tumour (pancreatic neuroendocrine neoplasm)

      CaseNotHPOs: HP:0030421; HP:0006770; HP:0100634; HP:0009711 (epididymal tumour, clear cell renal cell carcinoma, neuroendocrine neoplasm, retinal capillary hemangioma)

      CaseNotHPOFreeText: patient was recorded without CCRCC, and a pheochromocytoma and/or paraganglioma (neuroendocrine neoplasm), pancreatic endocrine tumour (or pancreatic neuroendocrine neoplasm), and a retinal hemangioblastoma, or a epididymal tumor

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Yes; see table S1 for VHL subject genetic and manifestation information.

      Variant: p.N78S (c.233A>G)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 93326 https://www.ncbi.nlm.nih.gov/clinvar/variation/93326/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transition

      CivicName: N78S (c.233A>G)

      MultipleGeneVariants: N/A

    4. 35 (45%) had missense variants,

      PatientID: 40

      KindredID: 40

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0100634 (Neuroendocrine neoplasm)

      CaseHPOFreeText: patient had pheochromocytoma/paraganglioma (recorded here as a neuroendocrine neoplasm)

      CaseNotHPOs: HP:0010797; HP:0006770; HP:0030405; HP:0009711; HP:0030421 (hemangioblastoma; clear cell renal cell carcinoma; pancreatic neuroendocrine neoplasm, retinal capillary hemangioma, Epididymal neoplasm)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Yes; see table S1 for VHL subject genetic and manifestation information.

      Variant: p.V84L (c.250G>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2236 https://www.ncbi.nlm.nih.gov/clinvar/variation/2236/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: V84L (c.250G>T)

      MultipleGeneVariants: N/A

    1. Case ReportA previously healthy 12-year-old boy was admitted to the hospital with vision loss, headache and dizziness over 1 month, which was aggravated in the last 5 days. Magnetic resonance imaging (MRI) of the head revealed a large (19.8 mm*18.5 mm*23.5 mm) irregular mass located in the suprasellar region abutting the left aspect of the optic chiasm. It appeared isointense on T1-weighted imaging (T1WI) and homogenously hyperintense on T2-weighted imaging (T2WI). After gadolinium administration, a well-circumscribed enhancing lesion was observed ( Figures 1A–E ). A small cerebellar lesion was also confirmed.Open in a separate windowFigure 1Brain MRI at admission (A–E). (A, B) Axial view (weighted sequence in T1, T2). (C–E) Sagittal, coronal, axial view after contrast injection, with the tumor marked. (F, G) Brain MRI after surgery. (H) Pancreatic cysts.The mother of the patient underwent surgery for HGB in the CNS (spine, cerebellum) and had pheochromocytomas and pancreatic cysts. On the basis of the mother’s medical history, clinical manifestations and imaging examinations, she was diagnosed with VHL disease, although she refused genetic sequencing. Therefore, the patient was examined for VHL disease before surgery. Sequencing of exon 1 of the VHL gene revealed a c.257C>T mutation (amino acid p.P86L). Ophthalmological examination revealed that the boundary of the optic disc in the left eye was pale and that there were no abnormalities in the right eye. Abdominal computed tomography (CT) was also performed to identify associated lesions and pancreatic cysts ( Figure 1H ). Based on these findings, the patient was definitely diagnosed with VHL syndrome.He underwent surgery to remove the tumor from the suprasellar region at our neurosurgical department, and histology confirmed the diagnosis of HGB ( Figures 2 and 3 ). The cerebellar lesion was not in the same surgical field as the suprasellar tumor and had no obvious mass effect, so they were not treated at the same time. Immunohistochemical analysis indicated that the Ki-67 index was approximately 2%. Furthermore, the neoplasm was positive for EGFR, NSE, CD34 and vimentin and negative for CK-pan, Pax-8, CD10, CD56, S-100, glial fibrillary acidic protein, inhibin, SSTR2, and epithelial membrane antigen ( Figure 2 ).Open in a separate windowFigure 2Representative postoperative pathological images. (A) Histologic examination of optic nerve HGB. (B–F) Immunohistochemical staining. (B) The proliferative fraction of tumor cells (Ki-67) was low, at approximately 2%. (C) CD-34 showed abundant blood vessels. Tumor cells showed positivity for EGFR (D), NSE (E) and vimentin (F).Open in a separate windowFigure 3Surgical pictures. Severe adhesion of the tumor and left optic nerve.The patient was transferred to the neurosurgery intensive care unit for postoperative monitoring and was ambulatory on postoperative day 1. Postoperative MRI ( Figures 1F, G ) demonstrated that the lesion in the suprasellar region was completely removed. He was discharged on postoperative day 7, and regular follow-up was performed. Unfortunately, the patient lost vision completely in the left eye after surgery.Go to:DiscussionHere, we reported a pediatric case of VHL disease presenting with HGBs in the optic nerve and cerebellum and pancreatic cysts. The diagnosis of VHL disease was confirmed by the identification of a familial germline mutation located in the first exon of the VHL gene (c.257C>T, p.P86L), which has been reported in the Human Gene Mutation Database and VHLdb databases (14, 15). To date, only 7 pediatric cases of sellar or suprasellar HGB (including optic nerve HGBs) have been reported ( Table 1 ). Regarding suprasellar HGBs, the pituitary stalk may be the site of origin in the vast majority of cases and, rarely, the intracranial portion of the optic nerve. In our case, we confirmed during surgery that the tumor was mainly located inside the optic nerve without a discernible tumor capsule and without the involvement of the leptomeninges. Given the rarity of suprasellar VHL-associated HGBs in childhood, we review the genotype-phenotype association of VHL disease, treatment, natural history and follow-up.

      PatientID: Case

      KindredID: 1

      Case: M, 12Y0M, Chinese (East Asian)

      DiseaseAssertion: VHL

      FamilyInfo: The mother of the patient underwent surgery for her hemangioblastoma in the CNS (spine, cerebellum) and had pheochromocytomas and pancreatic cysts. She was diagnosed with VHL disease, although she refused genetic sequencing.

      CasePresentingHPOs: HP:0006880; HP:0009711; HP:0001737 (cerebellar hemangioblastoma, retinal capillary hemangioma, pancreatic cysts)

      CaseHPOFreeText: Ophthalmological examination revealed that the boundary of the optic disc in the left eye was pale (hemangioblastoma) and that there were no abnormalities in the right eye. Unfortunately, the patient lost vision completely in the left eye after surgery. Abdominal CT was also performed to identify associated lesions and pancreatic cysts.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.P86L (c.257C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 182977 https://www.ncbi.nlm.nih.gov/clinvar/variation/182977/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: P86L (c.257C>T)

      MultipleGeneVariants: N/A

    1. his nephew

      PatientID: 31

      KindredID: 2

      Case: M, 27Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: The patient’s uncle (patient 34) was found to have the same variant, and had a malignant bilateral adrenal pheochromocytoma at age 31. The patient’s uncle was clinically diagnosed with VHL disease. The patient’s uncle died at an age of 46.

      CasePresentingHPOs: HP:0006748 (Adrenal pheochromocytoma)

      CaseHPOFreeText: The patient presented with a benign bilateral adrenal pheochromocytoma at age 24, and was originally diagnosed as a sporadic pheochromocytoma. This was later changed to a diagnosis of VHL disease through a retrospective screening.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R64P (c.191G>C)

      LegacyVariant: R64P (c.404G>C)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2226, https://www.ncbi.nlm.nih.gov/clinvar/variation/2226/

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: R64P(c.191G>C)

      MultipleGeneVariants: N/A

    1. In January 2013, a 74-year-old man was admitted to the Department of Neurosurgery at Yonsei University College of Medicine due to gait disturbance and tingling sensation of both legs. The patient was receiving beta-adrenergic antagonist and calcium channel blocker for arterial hypertension. He had lost his vision in the left eye due to cataract and amblyopia in the right eye due to retinitis which had occurred 30 years ago. The patient’s family history included a mother with a brain tumor of unconfirmed pathology, and both brothers with renal cell carcinoma with VHL gene mutation (Fig. 1). Serum carcinoembryonic antigen (CEA) was elevated to 11.78 ng/mL (normal value, 0.0 to 5.0 ng/mL). Although he had no history of diabetes mellitus, serum glucose level was 462 mg/dL and HbA1c was 11.7%, which led to a new diagnosis of diabetes mellitus, and initiation of insulin therapy. Whole spine magnetic resonance imaging (MRI) showed multiple enhancing nodular lesions of the spinal canal suspected leptomeningeal metastasis of unknown primary malignancy (Fig. 2A). Positron emission tomography whole body scan showed intense fludeoxyglucose uptake in a 3-cm-sized heterogeneous mass in the right kidney and focal mural thickening in the sigmoid colon (Fig. 2B and ​andC).C). Colonoscopy showed a 5-cm-sized ulceroinfiltrative lesion in the sigmoid colon and a polypoid lesion in the rectum. Subsequent histological examination confirmed the diagnosis of adenocarcinoma and high grade tubular adenoma with focal carcinoma in situ transformation, respectively. Incisional biopsy of the spinal mass was performed and the histological examination revealed hemangioblastoma.Open in a separate windowFig. 1.Pedigree presenting VHL gene mutation status and malignancy. Both brothers were diagnosed with renal cell carcinoma (RCC) with VHL gene mutation and one of his sons was confirmed VHL mutation without clinical manifestation.Open in a separate windowFig. 2.(A) Spine magnetic resonance imaging showed multiple enhancing nodular lesions of the spinal canal suspected leptomeningeal metastasis of unknown primary malignancy. Positron emission tomography computed tomography whole body scan showed a 3-cm enhancing heterogenous mass in the right kidney (arrow) (B), focal mural thickening with intense fludeoxyglucose uptake in the sigmoid colon (C).The patient underwent laparoscopic right partial nephrectomy, laparoscopic anterior resection of the sigmoid colon, and transanal endoscopic operation of the rectum. Histologic examination of the kidney revealed renal clear cell carcinoma (size, 1.9×1.7 cm), Fuhrman nuclear grade 3 (pT1aN0M0; stage I). In the sigmoid colon, gross pathology showed an ulcerofungating tumor (size, 4.2×3.6 cm) and 3 of 23 examined regional lymph nodes were metastasis. Subsequent histological examination confirmed typical features of colon adenocarcinoma (pT3N1bM0; stage IIIB). The rectum specimen was confirmed to be a 0.5-cm-sized well differentiated adenocarcinoma (pT1N0M0; stage I). The postoperative CEA level was 4.24 ng/mL.Regarding clinical decision processes, we initially suspected VHL disease. Subsequently, polymerase chain reaction (PCR) sequencing analysis of VHL gene showed p.Glu70Lys (c.208G > A) mutation in exon 1, confirming the diagnosis of VHL disease (Fig. 3). Result of endocrine hormonal evaluation for pheochromocytoma was within normal range.Open in a separate windowFig. 3.Polymerase chain reaction sequencing analysis of von Hippel–Lindau (VHL) gene showed a p.Glu70Lys (c.208G > A) mutation in exon 1, confirming the diagnosis of VHL disease.His two sons were screened for VHL disease using PCR sequencing analysis, and one progenity showed p.Glu70Lys (c.208G > A) mutation in exon 1, identical to his father. He underwent ophthalmic examination, abdomen-pelvic computed tomography scan and blood test for catecholamine metabolites, but there were no abnormal findings.After 3 weeks from discharge, he was admitted to hospital complaining of worsened gait disturbance and numbness of both soles. Brain MRI showed three enhancing lesions in bilateral cerebellar hemispheres and left cerebellar tonsil, suggesting hemangioblastomas (Fig. 4). The patient underwent gamma knife surgery in cerebellar lesions and palliative radiotherapy to T9-S1 spine with a total of 45 Gy in 25 daily fractions.Open in a separate windowFig. 4.Brain magnetic resonance imaging showed three enhancing lesions (arrows) in bilateral cerebellar hemispheres (A,B) and left cerebellar tonsil (C), suggesting hemangioblastomas.nAfter one month, follow-up imaging studies showed stable lesions in the brain and spine, but there was a newly developed hepatic metastasis in S6 and an enlarged aortocaval lymph node (Fig. 5). Concurrent serum CEA level increased to 19.54 ng/mL. The multidisciplinary team decided to perform surgery, laparoscopic wedge resection of liver and excision of aortocaval lymph node. Histologic examination of liver revealed a 1.6×1.2-cm-sized metastatic adenocarcinoma, moderately differentiated, clinically from colon. Pathologic finding indicated that the aortocaval lymph node was metastatic carcinoma, favoring renal clear cell carcinoma. He is on regular follow-up in the outpatient clinic and imaging studies show no evidence of recurrence and CEA level is stabilized within normal range.

      PatientID: None (Proband)

      KindredID: 1

      Case: M, 74Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo:Mother with a brain tumor of unconfirmed pathology, and both brothers (70Y and 79Y) with renal cell carcinoma with the same VHL gene mutation as this patient. One son (44Y) has the same mutation but was asymptomatic. Daughter (40Y) had a brain hemorrhage, niece had hematologic malignancy, nephew has pancreatic cancer; genetic information is unclear or not provided for these family members.

      CasePresentingHPOs: HP:0010797; HP:0000822; HP:0000518; HP:0000572; HP:0000646; HP:0000819; HP:0040276; HP:0006770; HP:0032118, HP:0030439; HP:0009713; HP:0002672; HP:0006880 (Hemangioblastoma, Hypertension, Cataract, Visual loss, Amblyopia, Diabetes mellitus, Adenocarcinoma of the colon, Clear cell renal cell carcinoma, Retinitis, Anal canal adenocarcinoma, Spinal hemangioblastoma, Gastrointestinal carcinoma, Cerebellar hemangioblastoma)

      CaseHPOFreeText: History of arterial hypertension and retinitis causing loss of vision in the left eye due to cataract and amblyopia in the right eye. History of retinal hemangioblastoma was presumed. Diagnosed with diabetes mellitus at the time of the study. MRI showed multiple enhancing nodular lesions of the spinal canal, later confirmed to be a hemangioblastoma. Whole body PET scan showed a heterogeneous mass in the right kidney and focal mural thickening in the sigmoid colon. Colonoscopy showed an ulceroinfiltrative lesion in the sigmoid colon and a polypoid lesion in the rectum subsequently confirmed as a adenocarcinoma and high grade tubular adenoma with focal carcinoma, respectively. Histologic examination of the kidney revealed renal clear cell carcinoma. In the sigmoid colon, gross pathology showed an ulcerofungating tumor and some regional lymph nodes were metastasis. The rectum specimen was confirmed to be a well differentiated adenocarcinoma. Brain MRI showed three enhancing lesions in bilateral cerebellar hemispheres and left cerebellar tonsil, suggesting hemangioblastomas. Histologic examination of liver revealed a metastatic adenocarcinoma. Pathologic finding indicated that the aortocaval lymph node was metastatic carcinoma, favoring renal clear cell carcinoma.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Figure 1

      Variant: p.E70K (c.208G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 43598, https://www.ncbi.nlm.nih.gov/clinvar/variation/43598/

      CAID: N/A

      gnomAD: 0.000004365, https://gnomad.broadinstitute.org/variant/3-10183739-G-A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: E70K (c.208G>A)

      MultipleGeneVariants: N/A

    1. 2655Fnt 658 G to T, Ala to Ser

      PatientID: 26

      KindredID: 6012

      Case: F, 55Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Another member of her family, (patient 25) presented with two radiologic pancreatic neuroendocrine tumors and had the same VHL gene mutation.

      CasePresentingHPOs: HP:0030405 (pancreatic endocrine tumour)

      CaseHPOFreeText: She presented with one radiologic pancreatic endocrine tumour.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.A149S (c.445G>T)

      LegacyVariant: p.A149S (c.658G>T)

      CaseProblemVariantFreeText: N/A

      ClinVar:127829 https://www.ncbi.nlm.nih.gov/clinvar/variation/127829/

      CAID: CA020355 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.445G%3ET

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: A149S (c.445G>T)

      MultipleGeneVariants: N/A

    2. 3939Fnt 713 G to A, Arg to GlnP, 15960

      PatientID: 39

      KindredID: 5960

      Case: F, 39Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Another member of his family (patient 38) has the same VHL mutation and presents with one radiologic pancreatic endocrine tumour.

      CasePresentingHPOs: HP:0030405 (pancreatic endocrine tumour)

      CaseHPOFreeText: He presented with one pathologic pancreatic endocrine tumour.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167Q (c.500G>A)

      LegacyVariant: p.R167Q (c.713G>A)

      CaseProblemVariantFreeText: N/A

      ClinVar: 2216 https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: CA020454 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.500G%3EA

      gnomAD: 0.000003977 https://gnomad.broadinstitute.org/variant/3-10191507-G-A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167Q (c.500G>A)

      MultipleGeneVariants: N/A

    1. The proband was a 34-year-old female with bilateral adrenal masses detected by ultrasonography. The patient did not have headaches, palpitations, predilection to sweating, nor other complaints. Physical examination was normal for her, including blood pressure. Family history of diseases was initially denied. Plasma cortisol, renin, angiotensin and aldosterone were all within normal values, except for urinary vanilmandelic acid (VMA), which was 114.2 µmol/day (normal range: 10–30 µmol/day). Computed tomography of the adrenal glands showed: (I) bilateral adrenal masses, both suggestive of PCCs; (II) a hepatic haemangioma in the left lobe (Figure 1A). Therefore, PCCs were highly suspected. The patient was given Prazosin regularly for 2 weeks, and laparoscopic bilateral partial adrenalectomy (LBPA) was finally performed. Later, histopathological examination confirmed that she had bilateral PCCs.

      PatientID: III-2

      KindredID: 1

      Case: F, 34Y0M, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: Four of 25 members of a Chinese family were found to harbour the p.Val98His mutation of the VHL gene with clinical VHL manifestations, and another 11 members of this family had this mutation but had no clinical manifestations of VHL. The patient’s brother (III-3), age 33, was found to have right adrenal PCC. The patient’s mother (II-1), age 61, was also found to have a right adrenal PCC. She also had a right RA (retinal angioma) approximately 10 years prior. Finally, the patient's cousin (III-4), male of age 31, had a right adrenal PCC 10 years prior and was found to have a left adrenal PCC.

      CasePresentingHPOs: HP:0006748; HP:0031207 (Adrenal pheochromocytoma, Hepatic hemangioma)

      CaseHPOFreeText: The patient was a 34-year-old Chinese female with bilateral adrenal masses detected by ultrasonography. Physical examination including blood pressure was normal. Computed tomography (CT) of the adrenal glands showed: (I) bilateral adrenal masses, both suggestive of PCCs; (II) a hepatic haemangioma in the left lobe (Figure 1A). A laparoscopic bilateral partial adrenalectomy (LBPA) was performed and histopathological examination confirmed bilateral PCCs.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.Y98H (c.292T>C)

      ClinVarID: 2223, https://www.ncbi.nlm.nih.gov/clinvar/variation/2223/

      gnomAD: 0.00001343, https://gnomad.broadinstitute.org/variant/3-10183823-T-C

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: Y98H(c.292T>C)

      MultipleGeneVariants: N/A

    1. Germline mutation profile of the VHL gene in von Hippel-Lindau disease and in sporadic hemangioblastoma

      PatientID: V5

      KindredID: V5

      Case: Sex Unknown, Age Unknown, French

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CasePresentingHPOs: N/A

      CaseHPOFreeText: Diagnosed with VHL Type 1.

      CaseNotHPOs: HP:0002666 (Pheochromocytoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.E55= (c.165G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 486721, https://www.ncbi.nlm.nih.gov/clinvar/variation/486721/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition; synonymous_variant

      CivicName: E55= (c.165G>A)

      MultipleGeneVariants: N/A

    2. PatientID: V374

      KindredID: V374

      Case: Sex Unknown, Age Unknown, French

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CasePresentingHPOs: N/A

      CaseHPOFreeText: Diagnosed with VHL Type 1.

      CaseNotHPOs: HP:0002666 (Pheochromocytoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.E70K (c.208G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 43598, https://www.ncbi.nlm.nih.gov/clinvar/variation/43598/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: E70K (c.208G>A)

      MultipleGeneVariants: N/A

    3. GroupID: V27

      DiseaseAssertion: VHL

      GroupSize: 3

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: French

      GroupAgeRange: N/A

      GroupInfo: Patients are grouped by family.

      FamilyInfo: 3/13 members of this family that underwent genetic testing had the same mutation.

      GroupPresentingHPOs: N/A

      GroupHPOFreeText: Diagnosed with VHL Type 1.

      GroupNotHPOs: HP:0002666 (Pheochromocytoma)

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: Likely 15300849

      SupplementalData: N/A

      Variant: p.L118P (c.353T>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 428807, https://www.ncbi.nlm.nih.gov/clinvar/variation/428807/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: L118P (c.353T>C)

      MultipleGeneVariants: N/A

    4. PatientID: V41

      KindredID: V41

      Case: Sex Unknown, Age Unknown, French

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CasePresentingHPOs: N/A

      CaseHPOFreeText: Diagnosed with VHL Type 1.

      CaseNotHPOs: HP:0002666 (Pheochromocytoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: Likely 15300849

      SupplementalData: N/A

      Variant: p.Y156* (c.468T>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA351756053, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.468T%3EG

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion; stop_gained

      CivicName: Y156* (c.468T>G)

      MultipleGeneVariants: N/A

    1. GroupID: None (25)

      DiseaseAssertion: VHL

      GroupSize: 1

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: This family group of 1 individual has clinically confirmed VHL disease and presented with retinal angiomatosis. This individual was found to harbor a c.499C>T variant causing a Arg167Trp missense variant (noted in the paper as c.712C>T due to Latif reference transcript being used).

      GroupPresentingHPOs: HP:0009711 (Retinal capillary hemangioma)

      GroupHPOFreeText: Patients within this family were found to have a clinical diagnosis of VHL disease. No VHL clinical phenotypes were explicitly stated other than retinal angiomatosis.

      GroupNotHPOs: N/A

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167W (c.499C>T)

      LegacyVariant: c.712C>T

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2218, https://www.ncbi.nlm.nih.gov/clinvar/variation/2218/

      CAID: N/A

      gnomAD: 0.000007954, https://gnomad.broadinstitute.org/variant/3-10191506-C-T?dataset=gnomad_r2_1

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    2. GroupID: None (15)

      DiseaseAssertion: VHL

      GroupSize: 2

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: This family group of 2 individuals have clinically confirmed VHL disease. These individuals were found to harbor a c.344A>G variant causing a His115Arg missense mutation (noted in the paper as c.557A>G due to Latif reference transcript being used).

      GroupPresentingHPOs: HP:0009711 (Retinal capillary hemangioma)

      GroupHPOFreeText: Patients within this family were found to have a clinical diagnosis of VHL disease. No VHL clinical phenotypes were explicitly stated other than retinal angiomatosis in at least one patient.

      GroupNotHPOs: N/A

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.H115R (c.344A>G)

      LegacyVariant: c.557A>G

      CaseProblemVariantFreeText: N/A

      ClinVarID: 664415, https://www.ncbi.nlm.nih.gov/clinvar/variation/664415/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: H115R (c.344A>G)

      MultipleGeneVariants: N/A

    1. PatientID: VII-4

      KindredID: 1

      Case: M, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Family’s roots were traced to the German town of Leipzig in Saxony, from whence the original 11 siblings had immigrated to America around 1670. The following report is a 2011 updated pedigree on the previously reported Tisherman kindred. At the time of this study, 43/49 patients have undergone mutational analysis revealing the VHL familial mutation (Y112H) and 35/49 patients have clinical VHL manifestations. Phenotypic cumulative lifetime risk was 71% for Pheo/PGL, 15% for hemangioblastoma, 33% for retinal angioma, 3% for renal cell carcinoma, and 3% for pancreatic cysts. 6 members of the family (all deceased; 4 males, 2 females) had suspected VHL and 2 (males) of these were suspected to have had Pheo/PGL.

      CasePresentingHPOs: HP:0002666; HP:0002668 (Pheochromocytoma, Paraganglioma)

      CaseHPOFreeText: Phenotype noted as: Pheo/PGL (unclear which of the two or if both apply to this patient).

      CaseNotHPOs: HP:0005584; HP:0009711 (Renal cell carcinoma, Retinal capillary hemangioma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 9201016; 8239848 (no genetic information; only phenotypes)

      SupplementalData: N/A

      Variant: p.Y112H (c.334T>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2222, https://www.ncbi.nlm.nih.gov/clinvar/variation/2222/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: Y112H (c.334T>C)

      MultipleGeneVariants: N/A

    2. PatientID: IV-11

      KindredID: 1

      Case: M, 56Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Family’s roots were traced to the German town of Leipzig in Saxony, from whence the original 11 siblings had immigrated to America around 1670. The following report is a 2011 updated pedigree on the previously reported Tisherman kindred. At the time of this study, 43/49 patients have undergone mutational analysis revealing the VHL familial mutation (Y112H) and 35/49 patients have clinical VHL manifestations. Phenotypic cumulative lifetime risk was 71% for Pheo/PGL, 15% for hemangioblastoma, 33% for retinal angioma, 3% for renal cell carcinoma, and 3% for pancreatic cysts. 6 members of the family (all deceased; 4 males, 2 females) had suspected VHL and 2 (males) of these were suspected to have had Pheo/PGL.

      CasePresentingHPOs: HP:0002666; HP:0006880 (Pheochromocytoma, Cerebellar hemangioblastoma)

      CaseHPOFreeText: Deceased (56Y). Cause of death:infarction of cerebellum. Right pheochromocytoma at autopsy.

      CaseNotHPOs: HP:0005584; HP:0009711; HP:0002668 (Renal cell carcinoma, Retinal capillary hemangioma, Paraganglioma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 9201016; 8239848 (no genetic information; only phenotypes)

      SupplementalData: N/A

      Variant: p.Y112H (c.334T>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2222, https://www.ncbi.nlm.nih.gov/clinvar/variation/2222/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: Y112H (c.334T>C)

      MultipleGeneVariants: N/A

    3. PatientID: VI-8

      KindredID: 1

      Case: M, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL (Asymptomatic)

      FamilyInfo: Family’s roots were traced to the German town of Leipzig in Saxony, from whence the original 11 siblings had immigrated to America around 1670. The following report is a 2011 updated pedigree on the previously reported Tisherman kindred. At the time of this study, 43/49 patients have undergone mutational analysis revealing the VHL familial mutation (Y112H) and 35/49 patients have clinical VHL manifestations. Phenotypic cumulative lifetime risk was 71% for Pheo/PGL, 15% for hemangioblastoma, 33% for retinal angioma, 3% for renal cell carcinoma, and 3% for pancreatic cysts. 6 members of the family (all deceased; 4 males, 2 females) had suspected VHL and 2 (males) of these were suspected to have had Pheo/PGL.

      CasePresentingHPOs: N/A

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0005584; HP:0009711; HP:0002666; HP:0002668 (Renal cell carcinoma, Retinal capillary hemangioma, Pheochromocytoma, Paraganglioma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 9201016; 8239848 (no genetic information; only phenotypes)

      SupplementalData: N/A

      Variant: p.Y112H (c.334T>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2222, https://www.ncbi.nlm.nih.gov/clinvar/variation/2222/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: Y112H (c.334T>C)

      MultipleGeneVariants: N/A

    4. PatientID: IV-16

      KindredID: 1

      Case: F, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL (Asymptomatic)

      FamilyInfo: Family’s roots were traced to the German town of Leipzig in Saxony, from whence the original 11 siblings had immigrated to America around 1670. The following report is a 2011 updated pedigree on the previously reported Tisherman kindred. At the time of this study, 43/49 patients have undergone mutational analysis revealing the VHL familial mutation (Y112H) and 35/49 patients have clinical VHL manifestations. Phenotypic cumulative lifetime risk was 71% for Pheo/PGL, 15% for hemangioblastoma, 33% for retinal angioma, 3% for renal cell carcinoma, and 3% for pancreatic cysts. 6 members of the family (all deceased; 4 males, 2 females) had suspected VHL and 2 (males) of these were suspected to have had Pheo/PGL.

      CasePresentingHPOs: N/A

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0005584; HP:0009711; HP:0002666; HP:0002668 (Renal cell carcinoma, Retinal capillary hemangioma, Pheochromocytoma, Paraganglioma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 9201016; 8239848 (no genetic information; only phenotypes)

      SupplementalData: N/A

      Variant: p.Y112H (c.334T>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2222, https://www.ncbi.nlm.nih.gov/clinvar/variation/2222/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: Y112H (c.334T>C)

      MultipleGeneVariants: N/A

    1. VHL_9Frozenc.482G>Ap.Arg161GlnM17AdrenalPCCB

      PatientID: VHL_9 (tumor ID)

      KindredID: 9

      Case: M, 17Y0M, Ethnicity Unknown

      DiseaseAssertion: Adrenal pheochromocytoma

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0006748 (Adrenal pheochromocytoma)

      CaseHPOFreeText: The patient had a benign adrenal pheochromocytoma at 17Y.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R161Q (c.482G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 182983, https://www.ncbi.nlm.nih.gov/clinvar/variation/182983/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R161Q (c.482G>A)

      MultipleGeneVariants: N/A

    2. VHL_19FFPEc.472C>Gp.Lys158ValMNAAdrenalPCCB

      PatientID: VHL_19 (tumor ID)

      KindredID: 19

      Case: M, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: Adrenal pheochromocytoma

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0006748 (Adrenal pheochromocytoma)

      CaseHPOFreeText: The patient had a benign adrenal pheochromocytoma at an unknown age.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.L158V (c.472C>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 567944, https://www.ncbi.nlm.nih.gov/clinvar/variation/567944/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: L158V (c.472C>G)

      MultipleGeneVariants: N/A

    3. VHL_4Frozenc.500G>Ap.Arg167GlnF30AdrenalPCCB

      PatientID: VHL_4 (tumour ID)

      KindredID: 4

      Case: F, 30Y0M, Ethnicity Unknown

      DiseaseAssertion: Adrenal Pheochromocytoma

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0006748 (Adrenal pheochromocytoma)

      CaseHPOFreeText: The patient had a benign adrenal pheochromocytoma at age 30.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167Q (c.500G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVar: 2216 https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: N/A

      gnomAD: 0.000003977 https://gnomad.broadinstitute.org/variant/3-10191507-G-A

      VariantEvidence:N/A

      MutationType: missense_variant;transition

      CivicName: R167Q (c.500 G >A)

      MultipleGeneVariants: N/A

    1. GroupID: 15

      DiseaseAssertion: VHL

      GroupSize: 2

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: Family of 2 VHL Type 1 affected individuals.

      GroupPresentingHPOs: HP:0010797; HP:0002666 (Hemangioblastoma, Pheochromocytoma)

      GroupHPOFreeText: CNS hemangioblastoma (1/2 affected members), pheochromocytoma (2/2 affected members).

      GroupNotHPOs: HP:0009711; HP:0002894; HP:0005584 (Retinal capillary hemangioma, Neoplasm of the pancreas, Renal cell carcinoma)

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167W (c.499C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2218, http://www.ncbi.nlm.nih.gov/clinvar/variation/2218

      CAID: N/A

      gnomAD: 0.000007954, http://gnomad.broadinstitute.org/variant/3-10191506-C-T

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    2. GroupID: 1

      DiseaseAssertion: VHL

      GroupSize: 5

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: Family of 5 VHL Type 1 affected individuals. Necropsy of the proband’s father, who died at 37 years from complications following emergency surgery for cerebellar HB. The necropsy showed many occult lesions related to VHL, including RC, multiple pancreatic and renal cysts, and cystadenoma of the epididymis. One non-symptomatic carrier in this family.

      GroupPresentingHPOs: HP:0010797; HP:0005584 (Hemangioblastoma, Renal cell carcinoma)

      GroupHPOFreeText: CNS hemangioblastoma (4/5 affected members) and renal carcinoma (2/5 affected members).

      GroupNotHPOs: HP:0009711; HP:0002894; HP:0002666 (Retinal capillary hemangioma, Neoplasm of the pancreas, Pheochromocytoma)

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: 32179488

      SupplementalData: N/A

      Variant: p.F76del (c.227_229del)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA357012, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.226_228del

      gnomAD: N/A

      VariantEvidence: NM_000551.3:c.226_228delTTC automaps to NM_000551.3:c.227_229delTCT due to redundancy.

      MutationType: inframe_deletion; deletion

      CivicName: F76del (c.227_229del)

      MultipleGeneVariants: N/A

    1. MSon3c.500G > Ap.Arg167Gln115–––––4

      PatientID: None

      KindredID: 11

      Case: M, 15Y0M, Korean

      DiseaseAssertion: VHL

      FamilyInfo: His father had the same mutation and presented with hemangioblastoma, renal cyst, retinal capillary hemangioma, and renal cell carcinoma. The grandmother and aunt were also suspected to have VHL mutations, but their DNA was not evaluated.

      CohortInfo: Thirteen unrelated patients with germline VHL mutations who were diagnosed with VHL disease were evaluated.

      CasePresentingHPOs: HP:0010797 (hemangioblastoma)

      CaseHPOFreeText: The patient developed a hemangioblastoma at age 15.

      CaseNotHPOs: HP:0001732, HP:0002666, HP:0000107, HP:0009711, HP:0005584 (abnormality of the pancreas, pheochromocytoma, renal cyst, retinal capillary hemangioma, renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167Q (c.500G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVar: 2216 https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: N/A

      gnomAD: 0.000003977 https://gnomad.broadinstitute.org/variant/3-10191507-G-A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167Q (c.500G>A)

      MultipleGeneVariants: N/A

    2. 9MProband3c.499C > Tp.Arg167Trp2B47–474747, M484

      PatientID: Proband

      KindredID: 9

      Case: M, 47Y0M, Korean

      DiseaseAssertion: VHL

      FamilyInfo: Niece presented with the same variant and CNS hemangioblastoma at 14Y, bilateral retinal hemangioblastoma at 26Y, RCC at 32Y, multiple pancreatic lesions (pancreatic cyst or tumor, unspecified) at 26Y and bilateral renal cysts at 32Y. Brother had a history of CNS hemangioblastoma but was not genetically confirmed.

      CohortInfo: Thirteen unrelated patients with germline VHL mutations who were diagnosed with VHL disease were evaluated.

      CasePresentingHPOs: HP:0010797; HP:0005584; HP:0001732; HP:0002666; HP:0000107; HP:0002315; HP:0002321 (Hemangioblastoma, Renal cell carcinoma, Abnormality of the pancreas, Pheochromocytoma, Renal cyst, Headache, Vertigo)

      CaseHPOFreeText: Presented with dizziness and headache, which he had experienced for the 3 months prior to examination. CNS hemangioblastoma at 47Y, RCC at 47Y, multiple pancreatic lesions (pancreatic cyst or tumor, unspecified) at 47Y, renal cyst at 48Y and pheochromocytoma at 47Y. Diagnosed with VHL Type 2B.

      CaseNotHPOs: HP:0009711 (Retinal capillary hemangioma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167W (c.499C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2218, https://www.ncbi.nlm.nih.gov/clinvar/variation/2218/

      CAID: N/A

      gnomAD: 0.000007954, https://gnomad.broadinstitute.org/variant/3-10191506-C-T?dataset=gnomad_r2_1

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    1. The c.640T>G germline variant, leading to a protein extension with the addition of a tail of 13 new amino acids, was found in a patient affected with a single pheochromocytoma at 67 years old.

      PatientID: 88

      KindredID: 88

      Case: Sex Unknown, 67Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0002666 (Pheochromocytoma)

      CaseHPOFreeText: Single pheochromocytoma.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Patient information pulled from Supplemental Table 1. There is additional tumour specific information in Supplemental Table 2.

      Variant: p.214Gext14 (c.640T>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA351756720

      gnomAD: N/A

      VariantEvidence: Membrane CA9 immunostaining was observed (supplemental table 1).

      MutationType: missense_variant;transversion; stop_lost

      CivicName: 214Gext14 (c.640T>G)

      MultipleGeneVariants: N/A

    2. PatientID: 82

      KindredID: 82

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: Assumed VHL

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0002666 (Pheochromocytoma)

      CaseHPOFreeText: Bilateral pheochromocytoma.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Patient information pulled from Supplemental Table 1b.

      Variant: p.A122V (c.365C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 1027087, https://www.ncbi.nlm.nih.gov/clinvar/variation/1027087/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: Membrane CA9 immunostaining was observed (supplemental table 1).

      MutationType: missense_variant;transition

      CivicName: A122V (c.365C>T)

      MultipleGeneVariants: N/A

    1. PatientID: None

      KindredID: 4589

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Two members of this family were diagnosed with VHL Type 2B. Both members presented with pheochromocytoma and one of these also presented with retinal angioma.

      CasePresentingHPOs: HP:0009711; HP:0002666 (Retinal capillary hemangioma, Pheochromocytoma)

      CaseHPOFreeText: Diagnosed with VHL Type 2B.

      CaseNotHPOs: HP:0010797; HP:0005584 (Hemangioblastoma; Renal cell carcinoma)

      CaseNotHPOFreeText: No CNS hemangioblastoma or RCC presentations.

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.C162F (c.485G>T)

      LegacyVariant: c.698G>T

      CaseProblemVariantFreeText: N/A

      ClinVarID: 43604, https://www.ncbi.nlm.nih.gov/clinvar/variation/43604/

      CAID: N/A

      gnomAD: 0.000003977, https://gnomad.broadinstitute.org/variant/3-10191492-G-T?dataset=gnomad_r2_1

      VariantEvidence: Variant reported as 689, G to T (Cys to Phe), assumed to be reported in legacy nomenclature (AF010238.1) and mapped to c.485G>T, p.Cys162Phe (NM_000551.3).

      MutationType: missense_variant;transversion

      CivicName: C162F (c.485G>T)

      MultipleGeneVariants: N/A

    1. Patient IV-2

      PatientID: IV-2

      KindredID: 1

      Case: F, 28Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Her paternal grandmother (II,2) did not carry the mutation. Her paternal grandfather (II-1) died age 42 from a cerebellar hemangioblastoma. Her uncle (III,1) did not harbour the mutation. The patient's father was the proband (III,2) and carried the mutation, presenting with clear cell renal cell carcinoma, hemangioblastoma of CNS, and retinal capillary hemangioblastoma. The mother did not carry the mutation. The patient had one unaffected sister (IV-1) and another who carried the mutation (IV-3) and had a history of pancreatic neuroendocrine tumor, hemangioblastoma of the CNS, and retinal capillary hemangioblastoma. Refer to pedigree in Figure 1.

      CasePresentingHPOs: HP:0006770, HP:0010797, HP:0009711, HP:0002666 (clear cell renal cell carcinoma, hemangioblastoma, retinal capillary hemangioma, pheochromocytoma )

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0030405 (pancreatic endocrine tumour)

      CaseNotHPOFreeText: No erythrocytosis; the hematocrit, red blood cells count and Epo levels were all normal.

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Yes supplemental data and figures. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5555745/bin/NIHMS881280-supplement-Supplemental_Data.pdf

      https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5555745/bin/NIHMS881280-supplement-Supplemental_Figures.pdf

      Variant: p.R200W (c.598C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2232 https://www.ncbi.nlm.nih.gov/clinvar/variation/2232/

      CAID: CA020510 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.598C%3ET

      gnomAD: 0.0002016 https://gnomad.broadinstitute.org/variant/3-10191605-C-T

      VariantEvidence: The patient was a heterozygous carrier of a single R200W and R161Q mutation (both on the same VHL allele).

      MutationType: missense_variant;transition

      CivicName:R200W(c.598C>T) and R161Q(c.482G>A)

      MultipleGeneVariants: Germline, VHL, c.482G>A (p.Arg161Gln)

    1. c.533 T>Cap.L178P

      GroupID: 26B

      Case: Sex, Age, Ethnicity unknown

      DiseaseAssertion: Unknown. Patient was pulled from either the VHL-Registry or NET- Registry and recorded with the given mutation. Symptoms or manifestations are not clear.

      FamilyInfo: N/A

      CasePresentingHPOs: N/A

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: HP:0030405 (pancreatic endocrine tumor)

      CasePreviousTesting: Patient was negatively tested for islet cell tumors (ICTs), or listed as pancreatic endocrine tumor.

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.L178P (c.533T>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 428795 https://www.ncbi.nlm.nih.gov/clinvar/variation/428795/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transition

      CivicName: L178P (c.533T>C)

      MultipleGeneVariants: N/A

    2. c.453 C>Gp.I151M

      GroupID: 16A

      Case: Sex, Age, Ethnicity unknown

      DiseaseAssertion: VHL

      FamilyInfo: None. The patient with the given mutation in this study was a VHL patient with pancreatic ICTs.

      CasePresentingHPOs: HP:0030405 (pancreatic endocrine tumor)

      CaseHPOFreeText: Patient has islet cell tumors (ICTs), or listed as pancreatic endocrine tumor.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.I151M (c.453C>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA351754364 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.453C%3EG

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: I151M (c.453C>G)

      MultipleGeneVariants: N/A

    3. c.500 G>Aap.R167Q

      GroupID: 25A

      DiseaseAssertion: VHL

      GroupSize: 4

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: 4 VHL patients with the given VHL mutation and manifestation were grouped in this study.

      GroupPresentingHPOs: HP:0030405 (pancreatic endocrine tumor)

      GroupHPOFreeText: Patients have islet cell tumors (ICTs), or listed as pancreatic endocrine tumor. Other clinical manifestations outside of a pancreatic neuroendocrine tumor are possible, but were not discussed.

      GroupNotHPOs: N/A

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167Q (c.500G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2216 https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: N/A

      gnomAD: 0.000003977 https://gnomad.broadinstitute.org/variant/3-10191507-G-A

      VariantEvidence: N/A

      MutationType: missense_variant; transition

      CivicName: R167Q (c.500G>A)

      MultipleGeneVariants: N/A

    1. 775 C>G 188 Leu>Val Missense 

      PatientID: 1695

      KindredID: 1695

      Case: Sex Unknown, 38Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CohortInfo: 44 patients with renal cell carcinoma who had known industrial exposure to trichloroethylene (TRI).

      CasePresentingHPOs: HP:0005584 (Renal cell carcinoma)

      CaseHPOFreeText: The patient had TRI exposure at age 27 and was diagnosed with renal cell carcinoma at age 38.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.L188V(c.562C>G)

      LegacyVariant: p.L188V(c.775C>G)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2225 https://www.ncbi.nlm.nih.gov/clinvar/variation/2225/?new_evidence=false

      CAID: N/A

      gnomAD: 0.00002121 https://gnomad.broadinstitute.org/variant/3-10191569-C-G

      VariantEvidence: The patient had LOH.

      MutationType: missense_variant;transversion

      CivicName: C162Y(c.486C>G) and L188V(c.562C>G) and P81S(c.241C>T) and F119L(c.357C>G)

      MultipleGeneVariants: Germline, VHL, c.241C>T (p.Pro81Ser), , Somatic, VHL, c.357C>G (p.Phe119Leu), Somatic, VHL, c.486C>G (Cys162Trp)

    2. 473 delTAT 87  Deletion 

      PatientID: 1654

      KindredID: 1654

      Case: Sex Unknown, 59Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CohortInfo: 44 patients with renal cell carcinoma who had known industrial exposure to trichloroethylene (TRI).

      CasePresentingHPOs: HP:0005584 (Renal cell carcinoma)

      CaseHPOFreeText: The patient had TRI exposure at age 25 and was diagnosed with renal cell carcinoma at age 59.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.V87Gfs*71(c.260_263del)

      LegacyVariant: p.V87Gfs*71(c.473_476del)

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID:CA2573320242 https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/by_caid?caid=CA2573320242

      gnomAD: N/A

      VariantEvidence: The patient had LOH.

      MutationType: minus_2_frameshift_variant

      CivicName: V87Gfs*71(c.260_263del)

      MultipleGeneVariants: Germline, VHL, c.241C>T (Pro81Ser)

    1. GroupID/ KindredID: F71

      Case: Sex unknown, 25Y0M, Spanish

      DiseaseAssertion: assumed VHL

      FamilyInfo: Noted a total of 2 affected family members. Other family member reported to have clear cell renal cell carcinoma (See Table 2).

      CasePresentingHPOs: HP:0009711; HP:0006880; HP:0002666 (retinal capillary hemangioma, cerebellar hemangioblastoma, pheochromocytoma)

      CaseHPOFreeText: Unilateral pheochromocytoma. Age of onset is 25Y0M.

      CaseNotHPOs: HP:0006770 (clear cell renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167Q (c.500G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVar: 2216 https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: N/A

      gnomAD: 0.000003977 https://gnomad.broadinstitute.org/variant/3-10191507-G-A

      VariantEvidence:N/A

      MutationType: missense_variant;transition

      CivicName: R167Q (c.500G>A)

      MultipleGeneVariants: N/A

    1. Twelve (75%) of 16 gene carriers were found to have ocular hemangioblastomas(Figure 1 and Figure 2), and 7 of these had bilateral disease (Table 1). The mean number of angiomas was 3.3. However, 1 individual had 22 angiomas, most of which were in an area of previous exudative retinal detachment (see the "Comment" section) (Table 2). Exclusion of this individual resulted in a mean number of angiomas of 2.1. Six eyes had optic disc angiomas (Figure 2). Six of the 19 eyes with ocular hemangioblastomas had visual acuity less than 20/40. Two patients had no light perception in one or both eyes. The average age at diagnosis of ocular angiomatosis was 37 years.

      PatientID: 15

      KindredID: 1

      Case: Sex Unknown, 29Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Significant family history. Pedigree follows 8 generations (see Figure 3). Family immigrated to Iowa, US, from Baden-Baden, Germany, in 1832; Likely sharing a common ancestor to the traced lineage of the Black Forest region of Germany.

      CasePresentingHPOs: HP:0002666 (pheochromocytoma)

      CaseHPOFreeText: 23Y at first pheochromocytoma; bilateral. Total of 2 pheochromocytoma tumors.

      CaseNotHPOs: HP:0009711; HP:0010797; HP:0005584 (retinal capillary hemangioma, hemangioblastoma, renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.Y98H (c.292T>C)

      LegacyVariant: p.Y98H (c.505T>C)

      CaseProblemVariantFreeText: N/A

      ClinVar: 2223 https://www.ncbi.nlm.nih.gov/clinvar/variation/2223/

      CAID: N/A

      gnomAD: 0.00001343 https://gnomad.broadinstitute.org/variant/3-10183823-T-C

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: Y98H (c.292T>C)

      MultipleGeneVariants: N/A

    1. 64-year-old man carrying the VHL gene mutation affected by simultaneous colon adenocarcinoma, renal clear cell carcinoma and adrenal pheochromocytoma.

      PatientID: None

      KindredID: 1

      Case: M, 75Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: The patient's brother had hypertension at age 77 and was found to carry the mutation. The patient's niece had breast cancer at age 33. Refer to Figure 4 for Pedigree.

      CasePresentingHPOs: HP:0006770, HP:0040276, HP:0006748, HP:0001407, HP:0002716, HP:0032618, HP:0100526, HP:0100598 (clear cell renal cell carcinoma, adenocarcinoma of the colon, adrenal pheochromocytoma, hepatic cysts, lymphadenopathy, renal necrosis, neoplasm of the lung, pulmonary edema)

      CaseHPOFreeText: Patient admitted at age 64 for intermittent abdominal pain, loss of appetite and constipation. He was found to have a solid tumor with areas of colliquative necrosis in the upper pole of the left kidney. The scans also showed distension of the colon with concomitant wall thickening, indicating possible neoplasia. Colonoscopy revealed a small polyp in the rectum-sigmoid junction, and a subsequent histological exam confirmed a diagnosis of adenocarcinoma. Both tumours were removed via a left hemicolectomy and left radical nephrectomy. A histologic examination of the kidney revealed renal clear cell carcinoma. Five years later, an abdominal CT scan revealed an adrenal pheochromocytoma in the right adrenal gland and multiple hepatic cysts in the right kidney and liver. The patient underwent a right adrenalectomy which found no convincing evidence of malignancy. At age 73 total body positron emission tomography (PET)-CT revealed the presence of several metastatic sites (both lungs, a 12×10 mm right sovraclaveare lymphadenopathy, and a 32×12 mm lesions at the mediastinum). An agobiopsy and cytologic broncholavage showed lymphocytes and epithelial cells with voluminous clear cytoplasm and large nuclei with prominent nucleoli, confirming the presence of metastatic lesions of clear cell renal carcinoma. In the same year, CT imaging revealed progression of pulmonary disease. One year later, a new CT scan revealed hepatic metastases and worsening of the mediastinal adenopathy and pulmonary metastases. At age 75, a new CT scan showed stable disease at various metastatic sites with minimal pleural effusion. Later in the month the patient was hospitalized for severe dyspnea, fatigue, and lower limb edema and died a few days later.

      CaseNotHPOs: HP:0009711 (retinal capillary hemangioma)

      CaseNotHPOFreeText: Fundoscopy revealed no vascular abnormalities of the retina.

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.P25L (c.74C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 93330 https://www.ncbi.nlm.nih.gov/clinvar/variation/93330/

      CAID: N/A

      gnomAD: 0.002988 https://gnomad.broadinstitute.org/variant/3-10183605-C-T

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName:P25L (c.74C>T)

      MultipleGeneVariants: N/A

    1. in seven of the asymptomatic family members.

      PatientID: IV-9

      KindredID: 1

      Case: F, Age Unknown, Turkish

      DiseaseAssertion: asymptomatic

      FamilyInfo: 17 family members from three generations were examined with the c.445G>T mutation and diagnosed with VHL type 2B. 11 individuals had clinical manifestations and 6 were asymptomatic carriers. One additional member (II-9) had passed before testing however were likely a carrier of the mutation. This patient’s father, aunt, grandmother and more, had the mutation. See Fig. 1 for full family pedigree.

      CasePresentingHPOs: N/A

      CaseHPOFreeText: None - described as a carrier of the p.A149S mutation, with no clinical manifestations. Their asymptomatic work up could have been due to the individual's young age

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.A149S (c.445G>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 127829 https://www.ncbi.nlm.nih.gov/clinvar/variation/127829/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: A149S (c.445G>T)

      MultipleGeneVariants: N/A

    2. Nine patients were diagnosed with pheochromocytoma. Lumbal spinal hemangioblastoma and pancreatic neuroendocrine tumor without pheochromocytoma were detected in one patient. In patients with pheochromocytoma, additional tumors, such as retinal angioma, renal cell carcinoma, pancreatic serous cystadenoma, and pancreatic neuroendocrine tumors were detected. All patients were diagnosed as VHL syndrome type 2B.

      PatientID: III-11

      KindredID: 1

      Case: M, 30Y0M, Turkish

      DiseaseAssertion: VHL

      FamilyInfo: 17 family members from three generations were examined with the c.445G>T mutation and diagnosed with VHL type 2B. 11 individuals had clinical manifestations and 6 were asymptomatic carriers. One additional member (II-9) had passed before testing however were likely a carrier of the mutation. This patient’s mother, sister, daughter, and more, had the mutation. See Fig. 1 for full family pedigree.

      CasePresentingHPOs: HP:0006748 (adrenal pheochromocytoma)

      CaseHPOFreeText: bilateral adrenal pheochromocytoma

      CaseNotHPOs: HP:0030393; HP:0009715 (endolymphatic sac tumor, papillary cystadenoma of the epididymis) 

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.A149S (c.445G>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 127829 https://www.ncbi.nlm.nih.gov/clinvar/variation/127829/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: A149S (c.445G>T)

      MultipleGeneVariants: N/A

    1. PatientID: 2

      KindredID: 6

      Case: 39Y0M, Sex and Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Recorded as a familial tumor; no other family info present

      CasePresentingHPOs: HP:0002666 (Pheochromocytoma)

      CaseHPOFreeText: Unilateral pheochromocytoma reported with an age at diagnosis of 39 years of age.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: RET, SDHB/SDHC/SDHD

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.S111C (c.331A>T)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 565557 https://www.ncbi.nlm.nih.gov/clinvar/variation/565557/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: S111C (c.331A>T)

      MultipleGeneVariants: N/A

    2. PatientID: 8

      KindredID: 9

      Case: 43Y0M, Sex and Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Recorded as a familial tumor; no other family info present

      CasePresentingHPOs: HP:0002666; HP:0002668 (Pheochromocytoma; Paraganglioma)

      CaseHPOFreeText: Left Pheochromocytoma and right paragangliomas were reported with an age at diagnosis of 43 years of age.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: RET, SDHB/SDHC/SDHD

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167Q (c.500G>A)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 2216 https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: N/A

      gnomAD: 0.000003977 https://gnomad.broadinstitute.org/variant/3-10191507-G-A

      VariantEvidence: N/A

      MutationType: missense_variant; transition

      CivicName: R167Q (c.500G>A)

      MultipleGeneVariants: N/A

    3. PatientID: 4

      KindredID: 8

      Case: 27Y0M, Sex and Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Recorded as a familial tumor; no other family info present

      CasePresentingHPOs: HP:0002666 (Pheochromocytoma)

      CaseHPOFreeText: Unilateral pheochromocytoma reported with an age at diagnosis of 27 years of age.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: RET, SDHB/SDHC/SDHD

      PreviouslyPublished: 19958924

      SupplementalData: N/A

      Variant: p.C162Y (c.485G>A)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 223225 https://www.ncbi.nlm.nih.gov/clinvar/variation/223225/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transition

      CivicName: C162Y (c.485G>A)

      MultipleGeneVariants: N/A

    1. PatientID: ccRCC-238

      KindredID: 238

      Case: M, 34Y0M, Ethnicity Unknown

      DiseaseAssertion: Clear Cell Renal Cell Carcinoma

      FamilyInfo: N/A

      CohortInfo: 240 ccRCC specimens.

      CasePresentingHPOs: HP:0006770 (clear cell renal cell carcinoma)

      CaseHPOFreeText:He was diagnosed with ccRCC at age 27. During the 84 month observation period he developed metastases in the contralateral kidney.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Supplementary table 1.

      Variant: p.S65L (c.194C>T)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 182975 https://www.ncbi.nlm.nih.gov/clinvar/variation/182975/

      CAID: N/A

      gnomAD: https://gnomad.broadinstitute.org/variant/3-10183725-C-T

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: S65L (c.194C>T)

      MultipleGeneVariants: N/A

    1. PatientID: 1 (case)

      KindredID: 1

      Case: M, 22Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: His mother and maternal grandfather had VHL disease, and his brother also had retinal angiomas

      CasePresentingHPOs: HP:0009711; HP:0006880; HP:0009713; HP:0005584; HP:0000107; HP:0030393 (retinal capillary hemangioma; cerebellar hemangioblastoma; spinal hemangioblastoma; renal cell carcinoma; renal cyst; endolymphatic sac tumor)

      CaseHPOFreeText: at 22Y had bilateral retinal angiomas and a 1-year history of truncal sensory disturbance. Neurologic examination showed mild left facial palsy and hypalgia at the level of T6-12, and audiometry disclosed left hearing disturbance by 23 decibels on pure tone average. Multiple lesions in the cerebellar hemispheres and in the spinal cord at the C5-6 and T11-12 levels were observed. A craniotomy for removal of the fourth ventricular tumor. Abdominal tumors disclosed bilateral renal cysts and left renal cell carcinoma. Additionally, a temporal bone tumor was discovered and removed, and consistent with ELST.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.Q73Rfs* (c.217delC)

      LegacyVariant: (c.930del)

      CaseProblemVariantFreeText: Written as: a single nucleotide deletion at nucleotide 930 in exon 1 (Reference sequence: AF010238.1), which caused a frame shift leading to a premature termination at codon 158. Two Cs at nucleotide 925 (*) were caused by compression, which was confirmed by other sequence data. This updates to NM_000551.3:p.Q73FS (c.215delC). c.215delC automaps to NM_000551.3:c.217delC

      ClinVarID: N/A

      CAID: CA432536422 https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.215delC

      gnomAD: N/A

      VariantEvidence: This mutation was detectable in all three samples, but the intensity of the wild type sequence was significantly lower in both tumor samples. The DNA from the hemangioblastoma showed loss of heterozygosity at both markers, whereas the DNA from the ELST showed loss of heterozygosity at D3S1259, but not at D3S1263

      MutationType: minus_1_frameshift_variant

      CivicName: Q73FS (c.215delC)

      MultipleGeneVariants: N/A

    1. PatientID: 16

      KindredID: 16

      Case: Sex Unknown, Age Unknown, Japanese

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0010797; HP:0005584 (Hemangioblastoma, Renal cell carcinoma)

      CaseHPOFreeText: CNS hemangioblastoma and RCC.

      CaseNotHPOs: HP:0009711; HP:0002666 (Retinal capillary hemangioma, Pheochromocytoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 10761708

      SupplementalData: N/A

      Variant: p.C77Sfs*82 (c.230del)

      LegacyVariant: codon 148 (c.443delG)

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA2573106239, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/by_caid?caid=CA2573106239

      gnomAD: N/A

      VariantEvidence: Nucleotide deletion (i.e. 443delG) coincides with legacy variant nomenclature (i.e. AF010238.1) and was mapped to c.230del (NM_000551.3).

      MutationType: minus_1_frameshift_variant; deletion

      CivicName: C77fs (c.230del)

      MultipleGeneVariants: N/A

    2. PatientID: 26

      KindredID: 26

      Case: Sex Unknown, Age Unknown, Japanese

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0009711; HP:0005584; HP:0010797 (Retinal capillary hemangioma, Renal cell carcinoma, Hemangioblastoma)

      CaseHPOFreeText: CNS hemangioblastoma, retinal angioma and RCC.

      CaseNotHPOs: HP:0002666 (Pheochromocytoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 10761708 (variants differ by one base pair; unclear which is accurate)

      SupplementalData: N/A

      Variant: p.G144Ffs*26 (c.429_439del)

      LegacyVariant: codon 214 (nucleotide 642 11 base pair deletion)

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA2573106247, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/by_caid?caid=CA2573106247

      gnomAD: N/A

      VariantEvidence: Given that all other variants are reported in legacy variant nomenclature (i.e. AF010238.1) this variant was mapped to c.429_439del (NM_000551.3).

      MutationType: minus_2_frameshift_variant; deletion

      CivicName: G144fs (c.429_439del)

      MultipleGeneVariants: N/A

    1. PatientID: N/A

      KindredID: 29982

      Case: Age unknown, Sex unknown, Ethnicity unknown

      DiseaseAssertion: assumed VHL

      FamilyInfo: Paper states: Germline, de novo (no clear confirmation)

      CasePresentingHPOs: HP:0010797 (hemangioblastoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Yes (Specifically Supp. Table S2)

      Variant: p.I151S (c.452T>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223215 https://www.ncbi.nlm.nih.gov/clinvar/variation/223215/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: I151S (c.452T>G)

      MultipleGeneVariants: N/A

    1. a duplication of 11 bp in the promoter region (c.-65_-55dup11)

      PatientID: 4

      KindredID: 4

      Case: F, 16Y0M, Ethnicity Unknown

      DiseaseAssertion: retinal capillary hemangioma

      FamilyInfo: N/A - family history was unremarkable. The paper states however that this patient inherited the duplication from a nonsymptomatic parent.

      CasePresentingHPOs: HP:0009711 (retinal capillary hemangioma)

      CaseHPOFreeText: a 16- year-old girl referred to this center for VHL testing due to an isolated retinal angioma

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 21463266

      SupplementalData: N/A

      Variant: c.–65_–55dup11

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 517152 https://www.ncbi.nlm.nih.gov/clinvar/variation/517152/

      CAID: N/A

      gnomAD: 0.00009581 https://gnomad.broadinstitute.org/variant/3-10183466-C-CTCCGCCCCGCG

      VariantEvidence: Quantitative Southern blot/real-time quantitative PCR analyses was normal in this patient. The 11 bp duplication is located within the promoter region of the VHL gene, in the vicinity of the main transcription initiation site, and within the binding site of several transcription factors. There is a possibility that the variant could alter transcription of the gene from the affected allele. Known polymorphic variant on the 3’UTR of the VHL gene, as well as the 5’UTR. PCR–RFLP assay approach showed that both alleles were expressed in apparently equimolar amounts (Fig. 2d) suggesting that the 11 bp duplication has no effect on expression of the affected allele.

      MutationType: 5_prime_UTR_variant

      CivicName: c.–65_–55dup11 AND 3' UTR alteration (c.*1149A>G)

      MultipleGeneVariants: Germline, VHL, c.*1149A>G

    1. GroupID: 174

      DiseaseAssertion: VHL

      GroupSize: N/A

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: VHL family found to have the c.543_544CA>TT (S111C) germline variant of the VHL gene. The number of family members included in this study is not specified.

      GroupPresentingHPOs: HP:0002666 (Pheochromocytoma)

      GroupHPOFreeText: Number of family affected members is not specified.

      GroupNotHPOs: N/A

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.S111C (c.330_331delinsTT)

      LegacyVariant: c.543_544CA>TT

      CaseProblemVariantFreeText:

      ClinVarID: N/A

      CAID: CA2573106137, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.330_331delinsTT

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: delins

      CivicName: S111C (c.330_331delinsTT)

      MultipleGeneVariants: N/A

    2. GroupID: 136

      DiseaseAssertion: VHL

      GroupSize: N/A

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: VHL family found to have a germline rearrangement of the VHL gene. The number of family members included in this study is not specified.

      GroupPresentingHPOs: N/A

      GroupHPOFreeText: Patients within this family were found to have a clinical diagnosis of VHL disease, however, the presenting phenotypes were not specified.

      GroupNotHPOs: HP:0002666 (Pheochromocytoma)

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: Likely previously published in PMID 32179488 (Kindred 14) but correspondence to a patient in this group is not confirmed.

      SupplementalData: N/A

      Variant: p.F76del (c.227_229del)

      LegacyVariant: c.439_441delTTC

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA357012, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.226_228del

      gnomAD: N/A

      VariantEvidence: NM_000551.3:c.226_228delTTC automaps to NM_000551.3:c.227_229delTCT due to redundancy

      MutationType: deletion; inframe_deletion

      CivicName: F76del (c.227_229del)

      MultipleGeneVariants: N/A

    3. GroupID: 7

      DiseaseAssertion: VHL

      GroupSize: N/A

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: VHL family found to have a germline rearrangement of the VHL gene. The number of family members included in this study is not specified.

      GroupPresentingHPOs: N/A

      GroupHPOFreeText: Patients within this family were found to have a clinical diagnosis of VHL disease, however, the presenting phenotypes were not specified.

      GroupNotHPOs: HP:0002666 (Pheochromocytoma)

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.Y175D (c.523T>G)

      LegacyVariant: c.736T>G

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA351756228, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.523T%3EG

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: Y175D (c.523T>G)

      MultipleGeneVariants: N/A

    4. GroupID: 93

      DiseaseAssertion: VHL

      GroupSize: Unknown

      GroupNumberMales: Unknown

      GroupNumberFemales: Unknown

      GroupEthnicity: Unknown

      GroupAgeRange: Unknown

      GroupInfo: This family group had a history of clinically confirmed VHL disease. Members of this family were found to harbour the c.481C>T variant causing a Arg161* stop gain (noted in the paper as c.694C>T due to Latif reference transcript being used). It was noted that this family had no history of pheochromocytoma's.

      GroupPresentingHPOs: N/A

      GroupHPOFreeText: Patients within this family were found to have a clinical diagnosis of VHL disease, however, the presenting phenotypes were not specified.

      GroupNotHPOs: HP:0002666 (pheochromocytoma)

      GroupNotHPOFreeText: It was noted that this family had no history of pheochromocytoma's.

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R161* (c.481C>T)

      LegacyVariant: p.R161* (c.694C>T)

      ClinVarID: 2217 https://www.ncbi.nlm.nih.gov/clinvar/variation/2217/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: stop_gained

      CivicName: R161* (c.481C>T)

      MultipleGeneVariants: N/A

    1. PMID: 23143947

      PatientID: DII1

      KindredID: D

      Case: M, 48Y0M, Chinese

      DiseaseAssertion: assumed VHL

      FamilyInfo: A 3-generation family is given with 3 confirmed members with this mutation. 2 other members had VHL-presenations however had not been tested for the gene.

      CasePresentingHPOs: HP:0006880; HP:0009711; HP:0001737; HP:0005584 (cerebellar hemangioblastoma, retinal capillary hemangioma, pancreatic cysts, renal cell carcinoma)

      CaseHPOFreeText: diagnosed with retinal capillary hemangioma at 24Y - adopted laser photocoagulation for left retinal hemangioblastoma at the age of 24, became blind 10 years later due to exudative retinal detachment. He had right cerebellar hemangioblastomas were removed at the age of 48, pancreatic cysts, and kidney disease at 48Y

      CaseNotHPOs: HP:0002666 (pheochromocytoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.F76del (c.226_228delTTC)

      LegacyVariant: p.F76del (c.439delTCT)

      CaseProblemVariantFreeText: NM_000551.3:c.226_228delTTC automaps to NM_000551.3:c.227_229delTCT

      ClinVarID: N/A

      CAID: CA357012 https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.226_228delTTC

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: inframe_deletion

      CivicName: F76del (c.227_2289delTCT)

      MultipleGeneVariants: N/A

    1. c.482G>A [p.Arg161Gln]

      PatientID: 2

      KindredID: 2

      Case: 18Y0M, Sex unknown, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: None

      CasePresentingHPOs: HP:0002666 (pheochromocytoma)

      CaseHPOFreeText: isolated bilateral pheochromocytoma first presenting at 18Y

      CaseNotHPOs: HP:0009715; HP:0005584; HP:0010797 (Papillary cystadenoma of the epididymis, renal cell carcinoma, hemangioblastoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: SDHB/SDHD

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R161Q (c.482G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 182983 https://www.ncbi.nlm.nih.gov/clinvar/variation/182983/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R161Q (c.482G>A)

      MultipleGeneVariants: N/A

    1. P1240MPCAtypicalVHL (p.Arg161X)NA10

      PatientID: P12

      KindredID: P12

      Case: M, 40Y0M, Ethnicity Unknown

      DiseaseAssertion: Pheochromocytoma

      FamilyInfo: N/A

      CohortInfo: Tumour samples and paired peripheral blood samples were obtained from 39 patients, consisting of 31 PCs, seven extra adrenal PGLs and one metastasis from a malignant PC previously presenting with a PC.

      CasePresentingHPOs: HP:0002666 (pheochromocytoma)

      CaseHPOFreeText: The patient had an atypical pheochromocytoma measuring 10 mm at age 40.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: RET/SDHB/SDHD

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R161* (c.481C>T)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 2217 https://www.ncbi.nlm.nih.gov/clinvar/variation/2217/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: stop_gained

      CivicName: R161* (c.481C>T)

      MultipleGeneVariants: N/A

    1. 16 different families

      PatientID: XV-I

      KindredID: 15

      Case: F, 24Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: No family history reported.

      CasePresentingHPOs: HP:0005584; HP:0000107; HP:0006880; HP:0009711; HP:0030405; HP:0001737; HP:0025247 (renal cell carcinoma, renal cysts, cerebellar hemangioblastoma, retinal capillary hemangioma, pancreatic endocrine tumor, pancreatic cysts, dermoid cyst)

      CaseHPOFreeText: Patient presented with renal cell carcinoma, renal cysts, cerebellar hemangioblastoma, retinal capillary hemangioma, pancreatic endocrine tumor, pancreatic cysts, dermoid cyst at age 24Y0M. Patient was diagnosed with VHL type 1 (see table 2).

      CaseNotHPOs: HP:0006748; HP:0002668 (adrenal pheochromocytoma; paraganglioma)

      CaseNotHPOFreeText: Patient negative for adrenal pheochromocytoma and paraganglioma.

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.S65W (c.194C>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 43597, www.ncbi.nlm.nih.gov/clinvar/variation/43597/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant, transversion

      CivicName: S65W (c.194C>G)

      MultipleGeneVariants: N/A

    2. 16 different families

      PatientID: VI-I

      KindredID: 6

      Case: M, 13Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: This kindred includes 3 patients: a father and his two children. The father, 55Y, had bilateral adrenal pheochromocytoma, along with his two children (13Y, 17Y).

      CasePresentingHPOs: HP:0006748 (adrenal pheochromocytoma)

      CaseHPOFreeText: Patient first presented with adrenal pheochromocytoma at age 13Y0M and was diagnosed with VHL type 2C (see table 2).

      CaseNotHPOs: HP:0001737; HP:000107; HP:0005584; HP:0002668; HP:0030405 (pancreatic cysts; renal cyst; renal cell carcinoma; paraganglioma; pancreatic endocrine tumor)

      CaseNotHPOFreeText: Patient negative for pancreatic cysts, pancreatic endocrine tumors, renal cell carcinoma, renal cysts, and paraganglioma.

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.L188del (c.562_564del)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA2573320489, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/by_caid?caid=CA2573320489

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: inframe_deletion

      CivicName: L188del (c.562_564del)

      MultipleGeneVariants: N/A

    1. PatientID: 1114

      KindredID: 236

      Case: Sex Unknown, Age Unknown, French

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CohortInfo: Families were recruited from the French VHL Registry. Enrolment in the study required identification of the germline VHL mutation (currently 85% of the families included in the French VHL Registry) and available data regarding renal phenotype. A total of 126 families fulfilled these criteria.

      CasePresentingHPOs: HP:0005584 (Renal cell carcinoma)

      CaseHPOFreeText: Severe renal disease (any solid lesion before 25 years of age, five or more solid lesions within one or two kidneys, or metastasis of renal cell carcinoma). Individual RCC phenotype is noted as positive.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: Paper states that this family is previously reported in PMID 12202531 and/or 9829912, where 1 and 4 individuals respectively with this mutation are reported. It is unclear if one or both of these previously reported groups are the same as the current family. These previously published individuals could alternatively correspond to family 345 or 379.

      SupplementalData: Phenotype and genotype information can be found in Supplementary Table S1.

      Variant: p.C162W (c.486C>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223227, https://www.ncbi.nlm.nih.gov/clinvar/variation/223227/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: C162W (c.486C>G)

      MultipleGeneVariants: N/A

    2. PatientID: 9579

      KindredID: 404

      Case: Sex Unknown, Age Unknown, French

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CohortInfo: Families were recruited from the French VHL Registry. Enrolment in the study required identification of the germline VHL mutation (currently 85% of the families included in the French VHL Registry) and available data regarding renal phenotype. A total of 126 families fulfilled these criteria.

      CasePresentingHPOs: HP:0005584 (Renal cell carcinoma)

      CaseHPOFreeText: Mild renal disease (simple renal cyst, multicystic kidney[s], small lesion [less than 10 mm in size] of unknown significance, or small solid tumor without indication for surgical removal [less than 30 mm in size]). Individual RCC phenotype is noted as positive.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 12202531

      SupplementalData: Phenotype and genotype information can be found in Supplementary Table S1.

      Variant: p.N78S (c.233A>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 93326, https://www.ncbi.nlm.nih.gov/clinvar/variation/93326/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: N78S (c.233A>G)

      MultipleGeneVariants: N/A

    3. GroupID: 8

      DiseaseAssertion: VHL

      GroupSize: 29

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: French

      GroupAgeRange: N/A

      GroupInfo: 29 members of this family were found to harbor the c.313A>C germline variant of the VHL gene. Members 572, 594, 1549, 1396, 580, 1394, 1392, 1399, 704 and 1550 had unknown renal involvement. Members 1153, 5098 and 1523 had no renal involvement. Members 1154, 6435 and 304 presented with mild renal disease. Members 574, 2419 and 1157 presented with moderate renal disease (RCC). Members 929, 654, 2993, 1398, 579, 571, 2281 and 2994 presented with severe renal disease (RCC). Families were recruited from the French VHL Registry. Enrolment in the study required identification of the germline VHL mutation (currently 85% of the families included in the French VHL Registry) and available data regarding renal phenotype. A total of 126 families fulfilled these criteria.

      GroupPresentingHPOs: HP:0009726; HP:0005584 (Renal neoplasm, Renal cell carcinoma)

      GroupHPOFreeText: Mild renal disease (simple renal cyst, multicystic kidney[s], small lesion [less than 10 mm in size] of unknown significance, or small solid tumor without indication for surgical removal [less than 30 mm in size]). Moderate renal disease (less than five solid lesions requiring surgical treatment, either unilateral or bilateral). Severe renal disease (any solid lesion before 25 years of age, five or more solid lesions within one or two kidneys, or metastasis of renal cell carcinoma).

      GroupNotHPOs: N/A

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: Paper states that this family is previously reported in PMID 12202531 and/or 9829912, where 9 and 14 individuals respectively with this mutation are reported. It is unclear if one or both of these previously reported groups are the same as the current family.

      SupplementalData: Phenotype and genotype information can be found in Supplementary Table S1.

      Variant: p.T105P (c.313A>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 496055, https://www.ncbi.nlm.nih.gov/clinvar/variation/496055/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: T105P (c.313A>C)

      MultipleGeneVariants: N/A

    1. GroupID: 17

      DiseaseAssertion: assumed VHL

      GroupSize: 16

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: 16 of the 573 patients discussed in this paper were found to have the following mutation belonging to 1 single family.

      GroupPresentingHPOs: HP:0005584; HP:0006880; HP:0009711 (renal cell carcinoma; cerebellar hemangioblastoma, retinal capillary hemangioma)

      GroupHPOFreeText: 6 patients had retinal angiomas diagnosed at a mean age of 24Y, 10 members had cerebellar hemangioblastomas diagnosed at a mean age of 41Y, and 2 had RCC diagnosed at a mean age of 34Y

      GroupNotHPOs: HP:0002666 (pheochromocytoma)

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Supplementary Table S1.

      Variant: p.G104Hfs*27 (c.309_310delTG)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: N/A

      CAID: CA658795178 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.309_310delTG

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: minus_2_frameshift_variant

      CivicName: G104FS (c.309_310delTG)

      MultipleGeneVariants: N/A

    2. GroupID: 44

      Case: Age unknown, Sex Unknown, Ethnicity Unknown

      DiseaseAssertion: assumed VHL

      FamilyInfo: 1 of the 573 patients discussed in this paper were found to have this variant. This mutation was found in 1 VHL family with a single patient.

      CasePresentingHPOs: HP:0006880; HP:0002666; HP:0009711; HP:0005584 (cerebellar hemangioblastoma; pheochromocytoma; retinal capillary hemangioma; renal cell carcinoma)

      CaseHPOFreeText: patient had all four of cerebellar hemangioblastoma; pheochromocytoma; retinal capillary hemangioma; renal cell carcinoma, with unknown ages

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Supplementary Table S1.

      Variant: c.341-2A>C

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA351753579 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.341-2A%3EC

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: splice_site_variant

      CivicName: Splice Site (c.341-2A>C) 

      MultipleGeneVariants: N/A

    3. GroupID: 34

      DiseaseAssertion: assumed VHL

      GroupSize: 7

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: 7 of the 573 patients discussed in this paper were found to have the following mutation belonging to 1 family 

      GroupPresentingHPOs: HP:0006880; HP:0005584 (cerebellar hemangioblastoma; renal cell carcinoma)

      GroupHPOFreeText: 3 had cerebellar hemangioblastomas diagnosed at mean age of 22Y, and 3 had RCC diagnosed at mean age of 48Y

      GroupNotHPOs: HP:0002666; HP:0009711 (pheochromocytoma; retinal capillary hemangioma)

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Supplementary Table S1.

      Variant: p.R177Dfs*25 (c.528delG) 

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA432423454 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.528delG

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: minus_1_frameshift_variant

      CivicName: R177fs (c.528del)

      MultipleGeneVariants: N/A

    1. GroupID: 1190

      DiseaseAssertion: VHL

      GroupSize: 19 GroupNumberMales: N/A GroupNumberFemales: N/A GroupEthnicity: German GroupAgeRange: N/A

      GroupInfo: Pennsylvanian pheochromocytoma family of German origin classified as a VHL Type 2A family according to the authors criteria (i.e. presenting with pheochromocytoma and without RCC or pancreatic cysts).

      GroupPresentingHPOs: HP:0002666; HP:0009711; HP:0010797 (Pheochromocytoma, Retinal capillary hemangioma, Hemangioblastoma)

      GroupHPOFreeText: 19 members affected with pheochromocytoma, 4 with retinal angioma, 1 with CNS hemangioblastoma.

      GroupNotHPOs: HP:0005584; HP:0001737 (Renal cell carcinoma, Pancreatic cysts)

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.T112H (c.334T>C)

      LegacyVariant: T547C (Y111H)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2222, https://www.ncbi.nlm.nih.gov/clinvar/variation/2222/

      CAID: N/A

      gnomAD: 0.00001343, https://gnomad.broadinstitute.org/variant/3-10183823-T-C

      VariantEvidence: Since this family was of German origin and was classified as a VHL Type 2A family, the authors chose to test 5 affected individuals (4612, 4613, 4796, 4799, 4801) and 7 unaffected individuals mutations at nucleotide 292. A novel migration fragment was identified in all 5 affected individuals in exon 1. None of the unaffected individuals showed this variant. DNA from patients 4612 and 4613 was amplified by PCR and subcloned resulting in the detection of the p.T112H (c.334T>C) variant. Sequencing in both directions confirmed this result. The other 3 affected individuals were not sequenced. Authors declare variant as “T547C (Y111H)” (assumed to follow legacy nomenclature) which was mapped to p.T112H (c.334T>C).

      MutationType: missense_variant;transition

      CivicName: T112H (c.334T>C)

      MultipleGeneVariants: N/A

    1. PMID: 7977367

      KindredID: 12

      DiseaseAssertion: VHL

      GroupSize: Unknown

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: This is a family belonging to prefigure No. 12. They are described as ‘VHL-affected’.

      GroupPresentingHPOs: HP:0005584 (renal cell carcinoma)

      GroupHPOFreeText: RCC-affected; other manifestations are possible but not clear.

      GroupNotHPOs: N/A

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.S65* (c.194C>A)

      LegacyVariant: p.136fs* (c.407C>A)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223161 https://www.ncbi.nlm.nih.gov/clinvar/variation/223161/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence:N/A

      MutationType: stop_gained

      CivicName: S65* (c.194C>A)

      MultipleGeneVariants: N/A

    1. PatientID: M

      KindredID: 1

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: Assumed VHL

      FamilyInfo: The patient's mutation was absent in the parents. Therefore the mutation is de-novo.

      CasePresentingHPOs: HP:0010797 (hemangioblastoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: HP:0009711, HP:0002894, HP:0002666, HP:0005584 (retinal capillary hemangioma, neoplasm of the pancreas, pheochromocytoma, renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.C162R (c.484T>C)

      LegacyVariant:p.C162R (c.697T>C)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 496067 https://www.ncbi.nlm.nih.gov/clinvar/variation/496067/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName:C162R (c.484T>C)

      MultipleGeneVariants: N/A

    1. PatientID: 7

      KindredID: A (III-4)

      Case: M, 41Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: The patient's father (II:3) died of cerebellar hemangioblastoma at age 39 and did not undergo genetic testing. The patient had two siblings who were healthy and did not undergo genetic testing. There was a strong VHL family history on the father's side of the family. Refer to Figure 1 for family pedigree and information regarding extended family members.

      CohortInfo: 10 VHL patients with renal cell carcinoma and a definite VHL family history.

      CasePresentingHPOs: HP:0010797, HP:0005584, HP:0009711, HP:0002666 (hemangioblastoma, renal cell carcinoma, retinal capillary hemangioma, pheochromocytoma)

      CaseHPOFreeText: Diagnosed with renal cell carcinoma and retinal angioma at age 24. He did not undergo surgery.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.V170D (c.509T>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 625256 https://www.ncbi.nlm.nih.gov/clinvar/variation/625256/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence:N/A

      MutationType: missense_variant;transversion

      CivicName: V170D (c.509T>A)

      MultipleGeneVariants: N/A

    1. In this case report, the authors describe an unusual case of initial collapse, seizures, and hypertensive crisis in a child who later was found to have multiple extraadrenal pheochromocytomas. Molecular diagnostics revealed a novel point mutation in the VHL gene (VHL nt. 406 T-->G). Only 7 months after the first lesions had been removed, a new paraganglioma developed in the contralateral periadrenal region.

      PatientID: 1 (case)

      KindredID: 1

      Case: M, 10Y4M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: No specific presentations were given for family history, however it was stated that the mutation was not found in the mother, yet the father’s DNA was not available for testing. The patient has 10 siblings, but all from a different father, none of which presented with a tumor or other severe disease known, and none was willing to be analyzed for the given mutation. It therefore cannot definitely differentiate between an inherited or a de novo mutation in this patient.

      CasePresentingHPOs: HP:0003712; HP:0006737; HP:0010302; HP:0002668 (Skeletal muscle hypertrophy, extraadrenal pheochromocytoma, spinal cord tumor, paraganglioma)

      CaseHPOFreeText: admitted to the hospital at 9Y with generalized seizures and marked hypertension; echocardiography showed muscular hypertrophy of the left ventricle; radiograph of the thorax revealed a round paramediastinal mass at the left side at the level of the fourth thoracic vertebra; MRI confirmed a thoracic structure (2 × 2 × 3 cm) and a tumor in the abdomen (2 × 3 × 5 cm) in the right suprarenal region; two separate tumors apart from the adrenal gland and the thoracic tumor were removed and found to be consistent with pigmented paragangliomas showing positivity for neuron-specific enolase, chromogranin A, and synaptophysin. 7 months after surgery he returned with tachycardia, vomiting, and hypertension and was found to have a new tumor in the 2 × 2 × 2 cm in the left periadrenal region; surgery found these to be three tumors in the proximity of the adrenal gland. 16 months after his first admission, the patient is in good health.

      CaseNotHPOs: HP:0010797; HP:0009711 (hemangioblastoma, retinal capillary hemangioma)

      CaseNotHPOFreeText: Physical examination revealed no other internal and no focal neurologic abnormalities. No café-au-lait-spots were present. No angiomatosis of the retina was present

      CasePreviousTesting: MEN2

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.S65A (c.193T>G)

      LegacyVariant: p.S65A (c.406T>G)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223160 https://www.ncbi.nlm.nih.gov/clinvar/variation/223160/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: S65A (c.193T>G)

      MultipleGeneVariants: N/A

    1. VHL-18c.233A > Gp.Asn78Ser[2]noA2Multiple–+–––

      PatientID: VHL-18

      KindredID: 18

      Case: Sex Unknown, Age Unknown, Italian

      DiseaseAssertion: VHL

      FamilyInfo: None.

      CohortInfo: Forty-three index cases were referred to the Service of Medical Genetics, Genova, for germline mutation analysis in the VHL gene between 1995 and 2008.

      CasePresentingHPOs: HP:0010797, HP:0001737 (hemangioblastoma, pancreatic cysts)

      CaseHPOFreeText: The patient had multiple hemangioblastomas.

      CaseNotHPOs: HP:0005584, HP:0002666, HP:0009711 (renal cell carcinoma, pheochromocytoma, retinal capillary hemangioma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.N78S(c.233A>G)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 93326 https://www.ncbi.nlm.nih.gov/clinvar/variation/93326/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType:missense_variant;transition

      CivicName: N78S(c.233A>G)

      MultipleGeneVariants: N/A

    1. 8aCerebellum443 ins TCT77insLIn frame insertionNegativeNegative 8bCerebellum443 ins TCT77insLIn frame insertionNegativeNegative8cCerebellum443 ins TCT77insLIn frame insertionNegativeNegative 8dCerebellum443 ins TCT77insLIn frame insertionNegativeNegative

      PatientID: 8 (tumour samples 8a, 8b, 8c, 8d)

      KindredID: 8

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CohortInfo: A total of 42 CNS haemangioblastoma samples were obtained from 31 patients, who were consecutively treated between 1993 and 1997 at the Department of Neurosurgery of the Freiburg University Medical Centre.

      CasePresentingHPOs: HP:0006880 (cerebellar hemangioblastoma)

      CaseHPOFreeText: Patient presented with 4 VHL-related tumors in the cerebellum (a,b,c,d).

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.C77_N78insL (c.230_231insTCT)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: p.77insL (c.443insTCT)

      ClinVarID: N/A

      CAID: CA2499214029 https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.230_231insTCT

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: inframe_insertion

      CivicName: C77_N78insL (c.230_231insTCT)

      MultipleGeneVariants: N/A

    1. two new mutations

      PatientID: 1

      KindredID: B

      Case: M, 35Y0M, Japanese

      DiseaseAssertion: Assumed VHL

      FamilyInfo: The patient’s family consisted of three members, but only the patient carried the missense variant NM_000551.3:c.227_229delTCT and developed symptoms; the patient’s parents did not. The authors of the paper assert that this was a novel mutation.

      CasePresentingHPOs: HP:0006880; HP:0011513 (Cerebellar hemangioblastoma, retinal angioma)

      CaseHPOFreeText: This patient developed brainstem hemangioblastoma and retinal angioma when he was 30 years old. Thereafter, he had no sign of recurrence for 5 years.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.F76del(c.227_229delTCT)

      LegacyVariant: p.F147del(c.440_442delTCT)

      ClinVarID: N/A

      CAID: CA357012, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/by_canonicalid?canonicalid=CA357012

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: inframe_deletion

      CivicName: F76del(c.227_229delTCT)

      MultipleGeneVariants: N/A

    1. c.464-3C > T1 (1%)163HBr, HBc, HBsc, RCC, Cr

      PatientID: None

      KindredID: 18

      Case: Sex Unknown, 63Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: N/A

      CohortInfo: All VHL mutation carriers of 18 years or older who underwent surveillance according to Dutch VHL guidelines between 1972 and 2012 in the VHL expertise centers University Medical Center Groningen (UMCG) and University Medical Center Utrecht (UMCU) were included. Patients with VHL-disease subtype 2C (pheochromocytoma only) were excluded.

      CasePresentingHPOs: HP:0009711; HP:0006880; HP:0009713; HP:0005584; HP:0000107 (Retinal capillary hemangioma, Cerebellar hemangioblastoma, Spinal hemangioblastoma, Renal cell carcinoma, Renal cyst)

      CaseHPOFreeText: VHL Type 1.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 24132471

      SupplementalData: Table 1

      Variant: c.464-3C>T

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 644549, https://www.ncbi.nlm.nih.gov/clinvar/variation/644549/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: splice_site_variant

      CivicName: Splice Region (c.464-3C>T)

      MultipleGeneVariants: N/A

    1. PMID: 10533077

      PatientID: 1

      KindredID: 1

      Case: Sex Unknown, Age Unknown, Jewish-Yemenite

      DiseaseAssertion: assumed VHL

      FamilyInfo: N/A - Four generation Jewish-Yemenite family in Israel but no details given; only give information for proband

      CasePresentingHPOs: HP:0002666; HP:0010797 (Pheochromocytoma, Hemangioblastoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.L178Q (c.533T>A)

      LegacyVariant: p.L178Q (c.746T>A)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 625260 https://www.ncbi.nlm.nih.gov/clinvar/variation/625260/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: L178Q (c.533T>A)

      MultipleGeneVariants: N/A

    1. c.191G>Cp.Arg64Pro135Unifocal (Adrenal)Syndromic

      PatientID: None

      KindredID: 6

      Case: Sex Unknown, 35Y0M, Southeast Asian

      DiseaseAssertion: VHL

      FamilyInfo: Positive family history for VHL.

      CohortInfo: This study retrospectively reviewed 124 consecutive patients with pheochromocytoma and paraganglioma that were seen in Singapore General Hospital, between 2005 and 2016.

      CasePresentingHPOs: HP:0006748 (Adrenal pheochromocytoma)

      CaseHPOFreeText: Presented with a unifocal adrenal pheochromocytoma at age 35.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: RET, VHL, NF1, SDHA, SDHB, SDHD, SDHAF2, TMEM127

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R64P(c.191G>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2226 https://www.ncbi.nlm.nih.gov/clinvar/variation/2226/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: R64P(c.191G>C)

      MultipleGeneVariants: N/A

    1. 48 F 39 B A VHL G644A G144Q

      PatientID: 48

      KindredID: 48

      Case: F, 39Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: None

      CohortInfo: Patients with apparently sporadic pheochromocytomas.

      CasePresentingHPOs: HP:0006748 (Adrenal pheochromocytoma)

      CaseHPOFreeText: The patient had a benign adrenal pheochromocytoma at age 39.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.G144E(c.431G>A) *Protein change is recorded improperly by the author

      LegacyVariant: p.G144E(c.644G>A)

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA351754206 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.431G%3EA

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName:G144E(c.431G>A)

      MultipleGeneVariants: N/A

    1. GroupID/ KindredID: VHL *

      DiseaseAssertion: assumed VHL

      GroupSize: 52

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: German

      GroupAgeRange: N/A

      GroupInfo: family 'VHL*' represents 14 VHL families for which a common founder has been identified in 16/52 (30%) of patients. 52 patients total with the given mutation belong to this cohort.

      GroupPresentingHPOs: HP:0009711; HP:0002666; HP:0010797 (retinal capillary hemangioma, pheochromocytoma, hemangioblastoma)

      GroupHPOFreeText: 26 patients recorded with retinal angiomas, 6 with hemangioblastomas of the central nervous system, and 41 pheochromocytomas. The authors state that this VHL 2A mutation confers a high risk of developing pheochromocytoma (with a 63% risk).

      GroupNotHPOs: N/A

      GroupNotHPOFreeText:  the authors state that this VHL 2A mutation confers a high risk of developing pheochromocytoma but a low risk of developing RCC (63% risk of pheochromocytoma but only a 4% risk of RCC).

      GroupPreviousTesting: N/A

      PreviouslyPublished: 1673491; 7759077

      SupplementalData: N/A

      Variant: p.Y98H (c.292T>C)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: p.Y98H (c.505T>C)

      ClinVarID: 2223 https://www.ncbi.nlm.nih.gov/clinvar/variation/2223/

      CAID: CA020246 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.292T%3EC

      gnomAD: 0.00001343 https://gnomad.broadinstitute.org/variant/3-10183823-T-C

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: Y98H (c.292T>C)

      MultipleGeneVariants: N/A

    1. c.500G>A p.Arg167Gln

      PatientID: VHL 1

      KindredID: VHL 1

      Case: Sex, Age, Ethnicity unknown

      DiseaseAssertion: hemangioblastoma

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0010797 (hemangioblastoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A - although likely duplicated in 24132471

      SupplementalData: Yes - See Supplementary Table 1

      Variant: p.R167Q (c.500G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2216 https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: N/A

      gnomAD: 0.000003977 https://gnomad.broadinstitute.org/variant/3-10191507-G-A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167Q (c.500G>A)

      MultipleGeneVariants: N/A

    1. The proband’s son (III.1) inherited his father’s germline VHL large deletion, and not the VHL p. Pro81Ser variant. In addition, the son (III.1) was found to have a hemizygous VHL c.340 + 5 G→C (IVS1 + 5) polymorphic variant, present on the allele inherited from his healthy mother (II.2). The son (III.1) was, therefore, clinically screened and found to have retinal and cerebellar hemangioblastomas as well as pancreatic cysts, typical lesions of VHL demonstrating that VHL cosegregated with, and is therefore caused by, the VHL large deletion and not the p.Pro81Ser variant.

      PatientID: III.1

      KindredID: 3

      Case: M, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: The patient inherited this mutation from his healthy mother (II:2). The patient's father (II:1) was the proband and had two VHL mutations, one of which he passed down to his son (exon 1-3 deletion). The father had hemangioblastomas and pancreatic cysts. The grandfather (I:1) had a P81S mutation but was otherwise healthy. Refer to pedigree in Figure 1-C.

      CohortInfo: Patients were registrants from the European-American Pheochromocytoma-Paraganglioma and German von Hippel-Lindau Disease Registries.

      CasePresentingHPOs: HP:0001737, HP:0006880, HP:0009711 (pancreatic cysts, cerebellar hemangioblastoma, retinal capillary hemangioma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: RET/SDHB/SDHD/SDHC

      PreviouslyPublished: May have been previously published in 11106358 and/or 7563486.

      SupplementalData: Yes, File 1 for methods.

      Variant: c.340+5G>C

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 36901 https://www.ncbi.nlm.nih.gov/clinvar/variation/36901/

      CAID: N/A

      gnomAD: 0.01119 https://gnomad.broadinstitute.org/variant/3-10183876-G-C

      VariantEvidence: Found to have a hemizygous VHL c.340+5G>C polymorphic variant, present on the allele inherited from his healthy mother.

      MutationType:splice_site_variant

      CivicName: Exon 1-3 Deletion and Splice Region (c.340+5G>C)

      MultipleGeneVariants: Germline, VHL, Exon 1-3 deletion

    1. 24 57 c.500G>T p.Arg167Leu x x x Familial Parents, sibling, and nephew Yes

      PatientID: 24

      KindredID: 24

      Case: Sex Unknown, 57Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Proband and father were found to carry the R167L (c.500G>T) germline mutation of the VHL gene. Father, siblings and nephew were analyzed but no phenotype information was provided for these family members. Genotype information for siblings and nephew was not provided.

      CasePresentingHPOs: HP:0002666; HP:0009711; HP:0010797 (Pheochromocytoma, Retinal capillary hemangioma, Hemangioblastoma)

      CaseHPOFreeText: Pheochromocytoma, retinal hemangioblastoma, CNS hemangioblastoma and pancreatic cyst or tumor.

      CaseNotHPOs: HP:0000107; HP:0006770; HP:0001732 (Renal cyst, Clear cell renal cell carcinoma, Abnormality of the pancreas)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Table S1, S2 and S3

      Variant: p.R167L (c.500G>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 526685, https://www.ncbi.nlm.nih.gov/clinvar/variation/526685/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: R167L (c.500G>T)

      MultipleGeneVariants: N/A

    2. PatientID: 328

      KindredID: 328

      Case: Sex Unknown, 41Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Family history noted but no details provided.

      CasePresentingHPOs: HP:0002666; HP:0000107; HP:0009711 (Pheochromocytoma, Renal cyst, Retinal capillary hemangioma)

      CaseHPOFreeText: Retinal hemangioblastoma, renal cystic lesion and pheochromocytoma.

      CaseNotHPOs: HP:0001732; HP:0010797; HP:0006770 (Abnormality of the pancreas, Hemangioblastoma, Clear cell renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Table S1, S2 and S3

      Variant: p.G93R (c.277G>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223174, https://www.ncbi.nlm.nih.gov/clinvar/variation/223174/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: G93R (c.277G>C)

      MultipleGeneVariants: N/A

    3. 263 50 c.36G>C p.Glu12Asp x n/d n/a -

      PatientID: 263

      KindredID: 263

      Case: Sex Unknown, 50Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Transmission of the variant could not be determined (n/d) since both parents and family history were not available.

      CasePresentingHPOs: HP:0010797 (Hemangioblastoma)

      CaseHPOFreeText: CNS hemangioblastoma.

      CaseNotHPOs: HP:0002666; HP:0006770; HP:0000107; HP:0001732; HP:0009711 (Pheochromocytoma, Clear cell renal cell carcinoma, Renal cyst, Abnormality of the pancreas, Retinal capillary hemangioma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Table S1, S2 and S3

      Variant: p.E12D (c.36G>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 419711, https://www.ncbi.nlm.nih.gov/clinvar/variation/419711/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: E12D (c.36G>C)

      MultipleGeneVariants: N/A

    1. 2015MbPCC––NoNoVHLc.500G>Abp.(Arg167Gln)Yesc

      PatientID: 20

      KindredID: 5

      Case: M, 15Y0M, Spanish

      DiseaseAssertion: VHL

      FamilyInfo: The patient had first-degree familial antecedents.

      CohortInfo: 36 pediatric patients with pheochromocytomas and/or parangangliomas.

      CasePresentingHPOs: HP:0002666 (pheochromocytoma)

      CaseHPOFreeText: The patient had a benign bilateral pheochromocytoma at age 15.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: The patient did not have any other tumours.

      CasePreviousTesting: RET, SDHA, SDHB, SDHC, SDHD, SDHAF2, TMEM127, MAX

      PreviouslyPublished: Yes source unknown.

      SupplementalData: N/A

      Variant: p.R167Q (c.500G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVar: 2216 https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: N/A

      gnomAD: 0.000003977 https://gnomad.broadinstitute.org/variant/3-10191507-G-A

      VariantEvidence:N/A

      MutationType: missense_variant;transition

      CivicName: R167Q (c.500 G >A)

      MultipleGeneVariants: N/A

    1. nt 475 T/A 88 Trp→Arg W88R Missense

      PatientID: 11

      GroupID/ KindredID: 11

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: asymptomatic

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0010797 (hemangioblastoma)

      CaseHPOFreeText: No other phenotypes. Patient’s tumor is stated to be primary asymptomatic

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.W88R (c.262T>A)

      LegacyVariant: p.W88R (c.475T>A)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 456577 https://www.ncbi.nlm.nih.gov/clinvar/variation/456577/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: W88R (c.262T>A)

      MultipleGeneVariants: N/A

    1. PatientID: 16

      KindredID: 6

      Case: F, 26Y0M, Indian

      DiseaseAssertion: VHL

      FamilyInfo: 1 other member of this family presented with the same variant. Patient 15 (Gen 2) presented with sympathetic paraganglioma and bilateral pheochromocytoma.

      CohortInfo: This retrospective study was conducted at Department of Endocrinology of a tertiary care health centre in Western India. Patients presented to our institute between 2008 and 2016 were included in the study. During the study period a total of 33 patients were suspected to have VHL syndrome based on clinical features among whom 31 patients were found to have mutation in VHL gene. All patients are of Asian Indian descent.

      CasePresentingHPOs: HP:0002666; HP:0001737 (Pheochromocytoma, Pancreatic cysts)

      CaseHPOFreeText: Bilateral pheochromocytoma.

      CaseNotHPOs: HP:0009711; HP:0010797; HP:0005584; HP:0030405; HP:0002668 (Retinal capillary hemangioma, Hemangioblastoma, Renal cell carcinoma, Pancreatic endocrine tumor, Paraganglioma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167Q (c.500G>A)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2216, https://www.ncbi.nlm.nih.gov/clinvar/variation/2216/

      CAID: N/A

      gnomAD: 0.000003977, https://gnomad.broadinstitute.org/variant/3-10191507-G-A

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167Q (c.500G>A)

      MultipleGeneVariants: N/A

    2. PatientID: 21

      KindredID: 11

      Case: M, 29Y0M, Indian

      DiseaseAssertion: VHL

      FamilyInfo: No relevant family history.

      CohortInfo: This retrospective study was conducted at Department of Endocrinology of a tertiary care health centre in Western India. Patients presented to our institute between 2008 and 2016 were included in the study. During the study period a total of 33 patients were suspected to have VHL syndrome based on clinical features among whom 31 patients were found to have mutation in VHL gene. All patients are of Asian Indian descent.

      CasePresentingHPOs: HP:0002666; HP:0010797; HP:0001737; HP:0005584 (Pheochromocytoma, Hemangioblastoma, Pancreatic cysts, Renal cell carcinoma)

      CaseHPOFreeText: Unilateral pheochromocytoma.

      CaseNotHPOs: HP:0009711; HP:0030405; HP:0002668 (Retinal capillary hemangioma, Pancreatic endocrine tumor, Paraganglioma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 32179488

      SupplementalData: N/A

      Variant: p.F76del (c.226_229del)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: N/A

      CAID: CA357012, https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/by_canonicalid?canonicalid=CA357012

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: inframe_deletion

      CivicName: F76del (c.227_229del)

      MultipleGeneVariants: N/A

    1. Case 2 A 41-year-old male patient was admitted to the Department of Ophthalmology and Endocrinology at the Affiliated Hospital of Zunyi Medical College between 24 September and 5 October 2014 due to blurred vision and elevated blood glucose level. During hospital admission, adrenal masses were identified on the left and right sides, and the patient was transferred to the Department of Urology on 23 October 2014. Vision was lost in the right eye due to a previous injury. The patient had hypertension and diabetes for >2 months. In September 2010, the patient underwent resection of the lesions in the right cerebellar hemisphere. Postoperative pathological analysis confirmed that the lesion was a hemangioblastoma. The mother of the patient was deceased, and the cause was unknown. The mother exhibited a brain tumor and coronary heart disease whilst alive. Multiple nodules were detected in the thyroid gland of the patient's eldest sister (II-1), but were not treated. The rest of the family members were healthy.

      PatientID: II-4

      KindredID: 2

      Case: M, 41Y0M, Chinese

      DiseaseAssertion: VHL

      FamilyInfo: The mother of the patient (I-2) was deceased, and the cause was unknown. The mother exhibited a brain tumor and coronary heart disease whilst alive. Multiple nodules were detected in the thyroid gland of the patient's eldest sister (II-1), but were not treated. The rest of the family members were healthy. One of the proband's sisters (II-2) and a brother (II-3) were found to carry the mutation. See Figure 2 for family pedigree.

      CasePresentingHPOs: HP:0000819, HP:0000822, HP:0010797, HP:0006748, HP:0002666, HP:0006744 (Diabetes mellitus, hypertension, hemangioblastoma, adrenal pheochromocytoma, pheochromocytoma, adrenocortical carcinoma)

      CaseHPOFreeText: A 41-year-old male patient was admitted due to blurred vision and elevated blood glucose level. Vision was lost in the right eye due to a previous injury. The patient had hypertension and diabetes for >2 months.CT scan showing nodules in the left and right adrenal glands. Low-density areas and calcification shadows are also observable. A contrast-enhanced scan revealed that the solid portion of the mass was markedly enhanced, but the cystic region was not enhanced. CT scan indicating cystic lesions or malacia in the right cerebellar hemisphere. The right occipital bone was altered following surgery.Postoperative abdominal CT indicating an irregular mass (~92×73×105 mm) in the right adrenal gland, which appeared to be pheochromocytoma and adrenal carcinoma. The left adrenal gland was altered following surgery. CT scan revealing multiple metastases in the left and right lungs.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: No abnormality was observed in the heart, lung or abdomen.

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167W (c.499C>T)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 2218 https://www.ncbi.nlm.nih.gov/clinvar/variation/2218/

      CAID: N/A

      gnomAD: 0.000007954 https://gnomad.broadinstitute.org/variant/3-10191506-C-T

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    1. PatientID: IV-6

      KindredID: 1

      Case: M, Age Unknown, Mexican

      DiseaseAssertion: Asymptomatic

      FamilyInfo: Family composed of six affected individuals and three diseased subjects (III-2, III-3, III-4) were studied. III-4 (proband) presented with a CNS hemangioblastoma and a retinal hemangioblastoma. The mother (II-1) developed cerebellar, medullar and bilateral retinal HBs; two maternal aunts (II-2 and II-3) were affected by retinal and CNS HBs and unspecified renal tumors, whereas his grandfather (I-2) had unspecified abdominal tumors. All these relatives died in their early 30s. A 35-year-old sister (III-2) demonstrated cerebellar HB, right eye retinal HB and renal cell carcinoma, whereas a 32-year-old brother (III-3) presented with cerebellar HB, right eye retinal HB, renal cell carcinoma and pheochromocytoma. A mutation-carrier nephew (IV-2) presented only atrophy of the retinal pigment epithelium. All of these family members were found to have the R82P (c.245G>C) germline VHL variant. Eighteen additional healthy relatives were analyzed and tested negative for the mutation.

      CohortInfo: Two families and one apparently non-familial case were studied at the Institute of Ophthalmology ‘Conde de Valenciana’ (Mexico City, Mexico).

      CasePresentingHPOs: N/A

      CaseHPOFreeText: Likely to be asymptomatic due to young age.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Family pedigree (Figure 1 (a))

      Variant: p.R82P (c.245G>C)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 526675, https://www.ncbi.nlm.nih.gov/clinvar/variation/526675/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: R82P (c.245G>C)

      MultipleGeneVariants: N/A

    1. R167W (28) 13 

      GroupID: None

      DiseaseAssertion: VHL

      GroupSize: 13

      GroupNumberMales:N/A

      GroupNumberFemales:N/A

      GroupEthnicity: N/A

      GroupAgeRange: ≥12

      GroupInfo: The thirteen patients were grouped by mutation.

      GroupPresentingHPOs: HP:0001732 (abnormality of the pancreas)

      GroupHPOFreeText: N/A

      GroupNotHPOs:N/A

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167W (c.499C>T)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 2218 https://www.ncbi.nlm.nih.gov/clinvar/variation/2218/

      CAID: N/A

      gnomAD: 0.000007954 https://gnomad.broadinstitute.org/variant/3-10191506-C-T

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    1. 181115N—N—NYYYYc.309_322del14aExon 1p.G104fs*231Frameshif

      PatientID: 18

      KindredID: 11

      Case: Sex Unknown, 15Y0M, Brazilian (Latino/Admixed American)

      DiseaseAssertion: VHL

      FamilyInfo: None

      CasePresentingHPOs: HP:0000107; HP:0009711; HP:0001737; HP:0010797 (renal cyst, retinal capillary hemangioma, pancreatic cyst, hemangioblastoma)

      CaseHPOFreeText: The patient at 15Y was confirmed with VHL. They had developed the above manifestations

      CaseNotHPOs: HP:0030405; HP:0002668, HP:0002666; HP:0005584 (pancreatic endocrine tumor, paraganglioma, pheochromocytoma, renal cell carcinoma)

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.G104Hfs*23 (c.309_322del14)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: N/A

      CAID: CA645524833 https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.309_322del14

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: minus_2_frameshift_variant

      CivicName: G104fs (c.309_322del14)

      MultipleGeneVariants: N/A

    1. A 65-year-old female was admitted to the outpatient clinic of the Urology Department, Peking University First Hospital, with a chief complaint of blood in her urine without pain in December of 2007. An abdominal computed tomography scan revealed bilateral RCC with multiple renal cysts as well as multiple pancreatic cysts (Fig. 1). Fifteen years ago, the patient was also found to have multiple cysts in both kidneys. She had no other clinical manifestations usually associated with VHL, such as the symptoms from CNS, retinal or adrenal involvement. Because of the diffuse nature of her bilateral kidney lesions, radical nephrectomy was not possible. The patient died 1 year after initial presentation.

      PatientID: N/A

      KindredID: 1

      Case: F, 66Y0M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Her son had a normal phenotype and was not found to carry the VHL mutation. Her daughter harboured the mutation but the PCR product from the patient was weaker than that from her daughter. The daughter presented with a CNS hemangioblastoma and died of complications at 37 years of age.

      CasePresentingHPOs: HP:0005584, HP:0005562, HP:0001737, HP:0000112 (renal cell carcinoma, multiple renal cysts, pancreatic cysts, nephropathy)

      CaseHPOFreeText: Fifteen years ago (age 50), the patient was also found to have multiple cysts in both kidneys. She was admitted to the outpatient clinic of the Urology Department, Peking University First Hospital, with a chief complaint of blood in her urine without pain in December of 2007. An abdominal computed tomography scan revealed bilateral RCC with multiple renal cysts as well as multiple pancreatic cysts. Furthermore, patient had severe bilateral kidney disease. The patient died at age 66 due to renal cell carcinoma.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: She had no other clinical manifestations usually associated with VHL, such as the symptoms from CNS, retinal or adrenal involvement.

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.S65W(c.194C>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 43597 https://www.ncbi.nlm.nih.gov/clinvar/variation/43597/

      CAID: CA020099 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.194C%3EG

      gnomAD: N/A

      VariantEvidence: The c.194C>G mutation rate was 18.8 ± 3.84% in the patient.

      MutationType: missense_variant;transversion

      CivicName:S65W(c.194C>G)

      MultipleGeneVariants: N/A

    1. 30266T>CLeu89ProSomatic mutation

      PatientID: 30

      KindredID: 30

      Case: Age, sex, ethnicity unknown

      DiseaseAssertion: hemangioblastoma

      FamilyInfo: N/A

      CasePresentingHPOs: HP:0010797 (hemangioblastoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.L89P (c.266T>A)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: 182979 https://www.ncbi.nlm.nih.gov/clinvar/variation/182979

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: This is a somatic varian found in the patient’s hemangioblastoma, found along with their germline c.194C>T

      MutationType: missense_variant;transversion

      CivicName: S65L (c.194C>T) AND L89P (c.266T>A)

      MultipleGeneVariants: Somatic, VHL, c.194C>T (p.Ser65Leu)

    1. 26 c.393CC>A p.Asn131fs p.N131fs Familial VHL

      GroupID/ KindredID: 26

      DiseaseAssertion: VHL

      GroupSize: 2

      GroupNumberMales: N/A

      GroupNumberFemales: N/A

      GroupEthnicity: N/A

      GroupAgeRange: N/A

      GroupInfo: 2 registrants belonging to the same family (26) have the germline VHL mutation.

      GroupPresentingHPOs: HP:0009711; HP:0000107; HP:0001737 (retinal capillary hemangioma, renal cyst, pancreatic cysts)

      GroupHPOFreeText: both 2 members had retinal hemangioblastomas, 1 had renal cysts, and 2 had pancreatic cysts

      GroupNotHPOs: HP:0010797; HP:0005584; HP:0002666 (hemangioblastoma, renal cell carcinoma, pheochromocytoma)

      GroupNotHPOFreeText: N/A

      GroupPreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.N131Kfs*28 (c.393_394delinsA)

      CaseProblemVariantFreeText: N/A

      LegacyVariant: N/A

      ClinVarID: N/A

      CAID: CA2573050931 https://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.393_394delinsA

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: delins

      CivicName: N131fs (c.393_394delinsA)

      MultipleGeneVariants: N/A

    1. PatientID: 3

      KindredID: 1

      Case: M, Age Unknown, German

      DiseaseAssertion: VHL

      FamilyInfo: This patient and his unaffected spouse (patient 4) had an affected son (patient 8) who carried both mutations and presented with pheochromocytoma. His son had two children; one that did not carry the mutation and was unaffected (patient 10) and another that carried both mutations but was disease free (patient 11). The patient's sister (patient 2) carried both mutations and presented with pheochromocytoma. She and her unaffected husband (patient 1) had four children; one died and presented with a pheochromocytoma, two (patient 5 and 7) had both mutations and presented with pheochromocytoma, and one child (patient 6) was unaffected and carried neither mutation. Patient 7 had a child (patient 9) who also carried both mutations and presented with pheochromocytoma. See Figure 3 for family pedigree.

      CasePresentingHPOs: HP:0002666 (pheochromocytoma)

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: 8772572, 7563486

      SupplementalData: N/A

      Variant: p.L188V (c.562C>G)

      LegacyVariant: p.L188V (c.775C>G)

      CaseProblemVariantFreeText: N/A

      ClinVar: 2225 https://www.ncbi.nlm.nih.gov/clinvar/variation/2225/

      CAID: CA020488 http://reg.clinicalgenome.org/redmine/projects/registry/genboree_registry/allele?hgvsOrDescriptor=NM_000551.3%3Ac.562C%3EG

      gnomAD: 0.00002121 https://gnomad.broadinstitute.org/variant/3-10191569-C-G

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: P81S (c.241C>T) and L188V (c.562C>G)

      MultipleGeneVariants: Germline, VHL, c.241C>T (p.Pro81Ser)

    1. PatientID: 9

      KindredID: 4

      Case: Sex Unknown, 55Y7M, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Positive VHL family history.

      CasePresentingHPOs: HP:0009711, HP:0010797, HP:0002666 (retinal capillary hemangioma, hemangioblastoma, pheochromocytoma)

      CaseHPOFreeText: At first presentation (age 54) the patient had a visual acuity of 20/100. The retinal angioma showed an exophytic growth pattern and was located in the superior disc margin. Before age 54, the patient had experienced hemangioblastoma of the central nervous system. Either at first presentation or on follow-up the patient presented with pheochromocytoma and hemangioblastoma of the CNS.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.L178Q(c.533T>A)

      LegacyVariant: p.L178Q(c.746T>A)

      CaseProblemVariantFreeText: N/A

      ClinVarID: 625260 https://www.ncbi.nlm.nih.gov/clinvar/variation/625260/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant;transversion

      CivicName: L178Q(c.533T>A)

      MultipleGeneVariants: N/A

    1. 1 (100)

      GroupID: 12

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL

      CohortInfo: a retrospective cohort study from an eye centre in Germany involved 148 VHL patients and their genetic data involved in retinal capillary hemangiomas.

      CasePresentingHPOs: HP:0009711 (retinal capillary hemangioma)

      CaseHPOFreeText: This individual patient was the only one discussed with this c.394C>T stop mutation. They had retinal hemangiomas; Other VHL manifestations were discussed but not per- variant.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Yes - Supplementary Table S1

      Variant: p.Q132* (c.394C>T)

      LegacyVariant: N/A

      GroupProblemVariantFreeText: N/A

      ClinVarID: 288749 https://www.ncbi.nlm.nih.gov/clinvar/variation/288749/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: stop_gained

      CivicName: Q132* (c.394C>T)

      MultipleGeneVariants: N/A

    2. 1 (50)

      GroupID: 39a

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL

      CohortInfo: a retrospective cohort study from an eye centre in Germany involved 148 VHL patients and their genetic data involved in retinal capillary hemangiomas.

      CasePresentingHPOs: HP:0009711 (retinal capillary hemangioma)

      CaseHPOFreeText: This was 1 of 2 patients discussed with this mutation. The individual had retinal hemangiomas; Other VHL manifestations were discussed but not per- variant.

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: Yes - Supplementary Table S1

      Variant: p.C162W (c.486C>G)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 223227 https://www.ncbi.nlm.nih.gov/clinvar/variation/223227/

      CAID: N/A

      gnomAD: N/A

      VariantEvidence: N/A

      MutationType: missense_variant; transversion

      CivicName: C162W (c.486C>G)

      MultipleGeneVariants: N/A

    1. 10cF““““RCC, HBA CNS, RCC, PanNET

      PatientID: 10c

      KindredID: 8

      Case: Sex Unknown, Age Unknown, Ethnicity Unknown

      DiseaseAssertion: VHL

      FamilyInfo: Family history was positive with 3 other members of unknown relation (10a, 10b, 10d) presenting with the mutation, with various phenotypes, however one member being asymptomatic as of age 3Y.

      CasePresentingHPOs: HP:0005584; HP:0010797; HP:0030405 (Renal Cell Carcinoma; Hemangioblastoma; Pancreatic endocrine tumor)

      CaseHPOFreeText: N/A

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: MEN1/ RET

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: p.R167W (c.499C>T)

      LegacyVariant: N/A

      CaseProblemVariantFreeText: N/A

      ClinVarID: 2218, https://www.ncbi.nlm.nih.gov/clinvar/variation/2218/

      CAID: N/A

      gnomAD: 0.000007954, https://gnomad.broadinstitute.org/variant/3-10191506-C-T?dataset=gnomad_r2_1

      VariantEvidence: N/A

      MutationType: missense_variant;transition

      CivicName: R167W (c.499C>T)

      MultipleGeneVariants: N/A

    1. Disease-associated genetic abnormalities were identified in all of the seven families examined.

      PatientID: nephew

      KindredID: C

      Case: M, 19Y0M, Chinese

      DiseaseAssertion: assumed VHL

      FamilyInfo: Family history is positive. Patient’s mother and aunt all had the splice mutation, two with CNS hemangioblastomas and one with adnexal papillary cystadenoma of probable mesonephric origin phenotypes.

      CasePresentingHPOs: HP:0010797; HP:0002666 (hemangioblastoma, pheochromocytoma)

      CaseHPOFreeText: Patient had hemangioblastoma of the CNS diagnosed at 19Y, and pheochromocytoma first at 14Y

      CaseNotHPOs: N/A

      CaseNotHPOFreeText: N/A

      CasePreviousTesting: N/A

      PreviouslyPublished: N/A

      SupplementalData: N/A

      Variant: c.340+5G>C

      LegacyVariant: c.553+5G>C

      CaseProblemVariantFreeText: N/A

      ClinVarID: 36901, https://www.ncbi.nlm.nih.gov/clinvar/variation/36901/

      CAID: N/A

      gnomAD: 0.01119 https://gnomad.broadinstitute.org/variant/3-10183876-G-C

      VariantEvidence: This variant’s large allele frequency means this is likely non disease causing (not deleterious).

      MutationType: splice_site_variant

      CivicName: Splice Region (c.340+5G>C)

      MultipleGeneVariants: N/A