A Prospective, Noninterventional Study to Evaluate the Impact of Emicizumab in the Management of Hemophilia A.

Background and objective Hemophilia A (HA) is a genetic bleeding disorder caused by a lack of factor VIII (FVIII) and is associated with frequent bleeding and joint damage. Traditional intravenous treatments for this condition are cumbersome and can lead to complications. Emicizumab, a bispecific monoclonal antibody, offers a promising subcutaneous alternative with potential safety and efficacy-related benefits. This study aimed to evaluate the impact of emicizumab prophylaxis on bleeding rates, joint health, functional activity, and quality of life (QoL) in patients with congenital HA. Methods A noninterventional, prospective observational study was conducted at the Assam Medical College, a tertiary care center in northeastern India, involving 40 patients with HA (PwHA), who were either on FVIII therapy or newly on emicizumab. Emicizumab was given subcutaneously at 3 mg/kg weekly for the first month, followed by 6 mg/kg every four weeks. Endpoints included changes in annual bleeding rate (ABR), Hemophilia Joint Health Score (HJHS), Functional Independence Score in Hemophilia (FISH), and QoL via European Quality of Life 5-Dimensions 5-Levels (EQ-5D-5L) and visual analog scale (VAS) scores at 24 weeks. Results At 24 weeks, HJHS improved from 12.8 to 4.8 (p<0.001), FISH from 27.5 to 30.6 (p<0.001), and ABR decreased from 11.36 to 0.195. Quality of life scores also improved (EQ-5D-5L index from 0.79 to 0.96, VAS from 72.18 to 92.75, both p<0.001). All 30 patients with target joints had resolved bleeds, and adherence to emicizumab was 100%. Conclusions Based on our findings, emicizumab significantly reduces bleeding, enhances joint health, and improves the quality of life in PwHA. It is associated with high adherence, suggesting its feasibility as a treatment, especially in resource-limited settings. However, long-term studies are needed to validate these results.