Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV000240172 | SCV000965663 | likely pathogenic | Hereditary thrombocytopenia and hematological cancer predisposition syndrome associated with RUNX1 | 2019-08-06 | reviewed by expert panel | curation | This in-frame deletion of exon 6 of RUNX1 does disrupt the critical RUNT homology DNA-binding domain which affects a crucial functional domain (PVS1_Strong). In addition, this deletion is absent from population databases (PM2). One proband shows a typical FPD/AML phenotype (PS4_supporting) and there is evidence of segregation of the deletion with disease in affected family members (PP1). In summary, this variant meets criteria to be classified as likely pathogenic. ACMG/AMP criteria applied, as specified by the ClinGen Myeloid Malignancy Variant Curation Expert Panel for RUNX1: PVS1_Strong, PM2, PP1, PS4_supporting. |
Labcorp Genetics |
RCV000240172 | SCV000299148 | likely pathogenic | Hereditary thrombocytopenia and hematological cancer predisposition syndrome associated with RUNX1 | 2016-04-27 | criteria provided, single submitter | clinical testing | This variant is a gross deletion of the genomic region encompassing exon 6 of the RUNX1 gene. This leads to an in-frame deletion, preserving the integrity of the reading frame. This deletion is not present in population databases. A similar deletion of RUNX1 exon 6 has been reported in the literature in an individual affected with thrombocytopenia and leukemia (PMID: 19946261). A missense substitution within this exon (p.Arg201Gln) is reported to be deleterious (PMID: 10508512, 21725049). This indicates that the Arginine residue, and therefore exon 6, is important for RUNX1 protein function. In summary, this variant is an in-frame gross deletion of exon 6 that has been reported in an affected individual and it has been shown to be important for RUNX1 protein function. For these reasons, it has been classified as Likely Pathogenic. |