Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV004821269 | SCV005442540 | pathogenic | von Willebrand disease type 2M | 2024-08-13 | reviewed by expert panel | curation | The NM_000552.5(VWF):c.3971G>C (p.Gly1324Ala) missense variant has been reported in at least one type 2M patient with VWF:RCo/VWF:Ag ratio of 0.44 (PMID: 12008946; PS4_supporting). The variant segregated with VWD type 2M through 3 affected meioses from this family (PP1; PMID:12008946). Another missense variant in the same codon has been reported in a patient with VWD type 2 Gly1324Ser, which has been classified likely pathogenic by the ClinGen VWD VCEP (PM5). The computational predictor REVEL gives a score of 0.719, which is above the ClinGen VWD VCEP threshold of >0.644 and predicts a damaging effect on VWF function (PP3). Platelet/GP1b binding assays performed with the Gly1324Ala recombinant mutant vWF expressed by COS-7 showed no binding indicating that this variant has a damaging effect on protein function (PMIDs: 12008946, 10845912; PS3). This variant is absent from gnomAD v4.1 (PM2_Supporting). In summary, this variant meets the criteria to be classified as Pathogenic for autosomal dominant VWD type 2M based on the ACMG/AMP criteria applied, as specified by the ClinGen VWD VCEP: PS3, PM2_supporting, PM5, PP1, PP3, PS4_supporting. |
Academic Unit of Haematology, |
RCV000086717 | SCV000118922 | not provided | not provided | no assertion provided | not provided |