Long-Term Joint Function and Quality of Life Following Emicizumab Prophylaxis in Severe Hemophilia A: A Single-Center Study From Goa, India

Background Hemophilia A (HA) is an established inherited bleeding condition caused by the lack of factor VIII, which causes recurrent joint hemorrhages and related adverse outcomes. Emicizumab, a bispecific monoclonal antibody, provides sustained prophylaxis, minimizing bleeding episodes and enhancing treatment adherence. This study assesses its impact on joint health, functional independence, and quality of life (QoL) in Indian patients with severe HA. Methodology This retrospective study, conducted at Goa Medical College, Goa, India, involved 20 patients with severe HA receiving emicizumab prophylactic therapy over a period of one year from January 2023 to January 2024. Outcomes were judged using the Hemophilia Joint Health Score (HJHS), annualized bleed rate (ABR), functional independence score in hemophilia (FISH), and the European Quality of Life 5-Dimension 5-Level (EQ-5D-5L) questionnaire for a period of one year. Ethical approval was secured for the study, and patient confidentiality was upheld. Results This study comprised 20 male participants (median age: 30.15 years), of whom 90% had severe HA and 55% had inhibitors present. Most (90%) participants were on an on-demand treatment protocol. At baseline, 90% of participants had target joints (mean: 4.7±2.51), and the mean ABR was 24.6±9.02. Over 12 months, HJHS decreased from 36.00 to 6.05 (p<0.001), FISH improved from 14.00 to 25.95 (p<0.001), and ABR dropped by 99.8% (median: 0.00, p<0.001). The EQ-5D-5L score significantly improved (p<0.001), indicating better QoL. Treatment adherence was 100%, with no adverse events (AEs). Conclusions Emicizumab demonstrated significant benefits in severe HA, including improved joint health, enhanced functional independence, and a better QoL over the course of 12 months. With 100% adherence and no AEs reported, it proves to be a feasible and well-tolerated prophylactic option. These findings support its long-term role in disease management, warranting further research in diverse patient populations.