ArticleTherapeutic advances in hematology2026
Twelve-month follow-up of a real-world, noninterventional study evaluating the impact of emicizumab on bleeding episodes, joint health, and quality of life in people with hemophilia A.
Article in Therapeutic advances in hematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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Who cites it
2 citing papers in PubMed.
- Plasma emicizumab concentrations and bleeding rates in children and adults with severe hemophilia A.Research and practice in thrombosis and haemostasis · 2026Observational
- Mechanism and kinetics study of MG1113, an anti-tissue factor pathway inhibitor antibody promoting hemostasis in hemophilia.Research and practice in thrombosis and haemostasis · 2026Article
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Authors and funding
6 authors.
Funding
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Abstract
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 ( 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.
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