Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV002254798 | SCV002525887 | pathogenic | Glanzmann thrombasthenia | 2022-05-17 | reviewed by expert panel | curation | The NM_000419.5(ITGA2B):c.957T>A (p.Tyr319Ter) variant in exon 11/30 is a nonsense variant predicted to lead to nonsense mediated decay in a gene in which loss-of-function is an established disease mechanism (PVS1). At least one patient (Patient GT12 in PMID:25373348) with this variant (compound heterozygous with c.2326_2331dup) displayed mucocutaneous bleeding and impaired aggregation with all agonists except ristocetin, which is highly specific for Glanzmann thrombasthenia. Additionally, alphaIIbbeta3 surface expression was severely reduced, as measured by flow cytometry. ITGA2B and ITGB3 were sequenced across all exons and intron/exon boundaries (PP4_strong). This variant is absent from gnomAD v2.1.1 (PM2_Supporting). In summary this variant meets criteria to be classified as Pathogenic for autosomal recessive Glanzmann Thrombasthenia based on the ACMG/AMP criteria applied, as specified by the ClinGen PD VCEP: PVS1, PP4_strong, PM2_supporting. (VCEP specifications version 2; date of approval 05/17/2022) |