A retrospective single-center study on the effectiveness and tolerability of emicizumab in patients with hemophilia a with and without inhibitors

BackgroundHemophilia A (HA) is an X-linked disorder due to deficient/defective coagulation factor VIII (FVIII), which causes excessive bleeding either traumatic or spontaneous. Frequent FVIII replacement is complicated by the development of inhibitors. The bispecific monoclonal antibody emicizumab, offers a novel approach by bridging activated factors IXa and X for effective hemostasis.Research design and methodsThis retrospective study analyzed emicizumab treatment outcomes in 73 patients with severe HA at a tertiary care center in Jammu and Kashmir, India. Data included demographic profiles, bleeding rates, joint health scores, and adverse events (AEs).ResultsEmicizumab demonstrated significant efficacy and safety in the study with median patient age of 14 years, with 17/73 patients having FVIII inhibitors. It resulted in a 100% reduction in annualized bleeding rate (ABR) for patients with inhibitors (from 20.7 to 0.0) and a 99.5% reduction for those without inhibitors (from 7.7 to 0.04). All inhibitor-negative patients showed ABR improvements and significant reduction in joint bleeds and resolution of all target joints. Tolerability was favorable with only one patient reporting a non-significant AE.ConclusionsEmicizumab prophylaxis effectively reduces ABRs and enhances joint health in severe HA patients, irrespective of inhibitor presence, providing a convenient, well-tolerated alternative to FVIII therapy.