Background: Hemophilia A (HA) management remains challenging in northeastern India due to limited access to specialized care. Emicizumab, a bispecific monoclonal antibody, offers prophylactic benefits for patients with or without factor VIII (FVIII) inhibitors. Objectives: The study aimed to assess the long-term effectiveness, safety, and patient-reported outcomes of emicizumab prophylaxis in people with HA with or without inhibitors. Design: This 12-month follow-up from a noninterventional, real-world observational study builds on previously published 6-month data and was conducted at the Department of General Medicine, Assam Medical College and Hospital, India. Methods: Forty pediatric patients with moderate-to-severe HA, including those with inhibitors, receiving emicizumab prophylaxis were followed at a treatment center. The outcomes assessed at baseline, 6 months, and 12 months included annualized bleeding rate (ABR), Hemophilia Joint Health Score (HJHS), Functional Independence Score in Hemophilia (FISH), European QoL-5 dimensional-5 level (EQ-5D-5L) score and visual analog scale (VAS). Subgroup analyses explored age- and inhibitor-status based differences. Data were analyzed using appropriate statistical tests to compare outcomes across time points and subgroups. Results: The mean ABR reduced significantly from 11.48 at baseline to 0.05 at 12 months ( p < 0.001), with all patients showing improvement. Joint and target joint bleeds were eliminated. HJHS improved from mean 12.0 ± 5.23 to 4.6 ± 3.13, and FISH increased from 27.5 ± 2.11 to 30.6 ± 1.39 (both p < 0.001). EQ-5D-5L index and VAS scores also showed significant gains. Notably, age-related disparities in ABR, evident with FVIII therapy, were no longer apparent after emicizumab therapy, highlighting its uniform effectiveness from early childhood. Conclusion: This 12-month follow-up study from northeastern India confirms the sustained efficacy and safety of emicizumab in reducing bleeds and improving outcomes in children with HA. The findings underscore its value in underserved settings, supporting broader implementation in similar healthcare environments.
Dutta et al. (Sun,) studied this question.