Total submissions: 3
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV000010960 | SCV004363660 | pathogenic | Hereditary factor VIII deficiency disease | 2024-02-02 | reviewed by expert panel | curation | The c.2215G>A (p.Glu739Lys) missense variant is absent from males in both gnomAD v2.1.1 and v3.1.1, meeting the PM2_Supporting criteria. The variant has a REVEL score of 0.614 (>0.6), meeting PP3 criteria. At least 13 patients with mild hemophilia A are reported in the literature and internal laboratory data meeting F8 phenotype criteria (PMID: 24452774, 23711237, 21166991, 8547094, 16972227), which meets the PS4_Very strong and PP4_Moderate criteria. This variant has been labeled as an inverse discrepant variant, where the one-stage assays show lower factor VIII levels than the two-stage, or chromogenic, assays (EAHAD database; PMID: 32232366). There were multiple related individuals across 6 different families reported that were genotyped, but their specific relation was not stated, so the PP1 was capped at the moderate weight to be conservative (PMID: 24452774). In-vitro assays in COS-1 cells show FVIII-Glu739Lys results in lower FVIII:C in the mild range, as observed in patients with this variant and hemophilia A (PMID: 30997536). In summary, this variant meets criteria to be classified as pathogenic. ACMG/AMP criteria applied, as specified by the Coagulation Factor Deficiency Variant Curation Expert Panel for F8: PS4_Very strong, PS3, PP1_Moderate, PP4_Moderate, PP3, PM2_Supporting. |
OMIM | RCV000010960 | SCV000031187 | pathogenic | Hereditary factor VIII deficiency disease | 1995-01-01 | no assertion criteria provided | literature only | |
Prevention |
RCV004745152 | SCV005362704 | likely pathogenic | F8-related disorder | 2024-04-03 | no assertion criteria provided | clinical testing | The F8 c.2215G>A variant is predicted to result in the amino acid substitution p.Glu739Lys. This variant (aka. p.Glu720Lys) has been reported in multiple individuals with hemophilia A (Schwaab et al. 1995. PubMed ID: 8547094; Factor VIII (F8) Gene Variant Database: https://www.factorviii-db.org/index.php). This variant is reported in 0.0092% of alleles in individuals of European (Non-Finnish) descent in gnomAD. This variant is interpreted as likely pathogenic. |