Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV004577685 | SCV005061693 | pathogenic | Glanzmann thrombasthenia | 2024-02-20 | reviewed by expert panel | curation | The c.1423C>T (p.Gln475Ter) variant is a nonsense variant predicted to cause a premature stop codon in biologically-relevant-exon 14/30 and is predicted to lead to nonsense mediated decay in a gene in which loss-of-function is an established mechanism (PVS1; PMID:31980526). At least one patient (Patient 8 in PMID:36672149) with this variant displayed mucocutaneous bleeding and impaired aggregation with all agonists except ristocetin, which is highly specific for Glanzmann thrombasthenia. Additionally, αIIbβ3 surface expression was absent (<25%), as measured by flow cytometry. However, ITGA2B and ITGB3 were not reported to be sequenced across all exons and intron/exon boundaries (PP4_moderate). The same individual was homozygous for the variant and confirmed in trans (0.5 PM3 points, PMID:36672149). Total points: 0.5 (PM3_supporting). This variant is absent from gnomAD v.2.1.1 (PM2_supporting). In summary, this variant meets the 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, PM3_supporting, PM2_supporting, PP4_moderate. (VCEP specifications version 2; date of approval 02/20/2024) |