Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV003448648 | SCV004176253 | likely pathogenic | Hereditary thrombocytopenia and hematologic cancer predisposition syndrome | 2023-12-09 | reviewed by expert panel | curation | NM_001754.5(RUNX1):c.592G>T (p.Asp198Tyr) is a missense variant. This variant is completely absent from all population databases with at least 20x coverage for RUNX1 in gnomAD v2.1.1 and v3.1.2 (PM2_supporting). This variant affects one of the 13 hotspot residues established by the MM-VCEP for RUNX1 (PM1). This missense variant has a REVEL score ≥ 0.88 (0.964) (PP3). Yeast assays showed significant decreased DNA binding, CBFbeta binding and promoter activation indicating that this variant impacts protein function (PMIDs 17290219, 23848403)(PS3_Supporting). This variant has been reported in 3 probands of a family meeting RUNX-1 phenotypic criteria (FPD and/or AML) (PMID 11675361) (PS4_supporting, PP1_supporting). In summary, the clinical significance of this variant is likely pathogenic. ACMG/AMP criteria applied, as specified by the Myeloid Malignancy Variant Curation Expert Panel for RUNX1: PM1, PP1_supporting, PP3, PM2_supporting, PS3_supporting, PS4_supporting. |