AOZORA study: 3-year interim analysis of safety and joint health in pediatric people with hemophilia A receiving emicizumab prophylaxis

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Background: Recurrent joint bleeding in people with hemophilia A (PwHA) can cause hemophilic arthropathy, resulting in limited movement and chronic pain. Emicizumab is a bispecific monoclonal antibody bridging activated factor (F)IX and FX to substitute for deficient activated FVIII in PwHA, thereby improving hemostasis. Objectives: This 3-year interim analysis of the ongoing, open-label, phase IV AOZORA study (jRCT1080224629) analyzes medium-term safety and joint health in pediatric PwHA without FVIII inhibitors receiving emicizumab. Methods: PwHA aged <12 years with severe hemophilia A without FVIII inhibitors were eligible. Participants entered AOZORA as emicizumab-naïve or having previously initiated emicizumab during the HOHOEMI study. Endpoints included safety, and joint health, as assessed by magnetic resonance imaging and Hemophilia Joint Health Score (HJHS). Participants will receive emicizumab for 6 years. Results: A total of 30 male PwHA were enrolled. Data cutoff was the last day of week 145 for each participant. Median (range) age was 4.2 (0.7-11.1) years, and 27 of the 30 (90.0%) had received prior FVIII prophylaxis. The emicizumab safety profile was confirmed. No thrombotic events/microangiopathies occurred. All joints with synovial hypertrophy and hemosiderin resolved or improved by week 145. HJHS remained at 0 from week 1 to week 145 for 18 (66.7%) participants; overall, there was no worsening trend in HJHS over time. Model-based annualized bleeding rate (95% CI) for treated bleeds was 3.6 (2.04-6.46) prior to emicizumab and 0.8 (0.47-1.22) after receiving emicizumab. Conclusion: Emicizumab is well tolerated and appears to maintain or improve joint health in pediatric PwHA.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.