Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV002254822 | SCV002525915 | likely pathogenic | Glanzmann thrombasthenia | 2022-04-15 | reviewed by expert panel | curation | The NM_000212.3(ITGB3):c.1388G>C (p.Cys463Ser) missense variant has been observed in at least one GT patient (Patient 7 in PMID: 34267460), whom displayed mucocutaneous bleeding and impaired aggregation with all agonists except ristocetin, which is highly specific for Glanzmann thrombasthenia. Additionally, alphaIIbbeta3 surface expression was reduced to 0.5-1.7%, as measured by flow cytometry (PP4_strong). The variant was been reported to segregate with Glanzmann thrombasthenia in Patient 7 plus one affected family member, both with the homozygous Cys463Ser genotype. (PM3_supporting, PP1; PMID: 34267460). This variant is absent from gnomAD v2.1.1 (PM2_Supporting). The computational predictor REVEL gives a score of 0.898, which is above the ClinGen PD VCEP threshold of >0.7 and predicts a damaging effect on function (PP3). In summary this variant meets criteria to be classified as Likely Pathogenic for autosomal recessive Glanzmann Thrombasthenia based on the ACMG/AMP criteria applied, as specified by the ClinGen PD VCEP: PP4_strong, PP1, PP3, PM2_supporting, PM3_supporting. (VCEP specifications version 2.1) |