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

Abstract

Background: Hemophilia 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 methods: This 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).

Results: Emicizumab 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.

Conclusions: Emicizumab 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.