ArticleResearch and practice in thrombosis and haemostasis2025
AOZORA study: 3-year interim analysis of safety and joint health in pediatric people with hemophilia A receiving emicizumab prophylaxis.
Article in Research and practice in thrombosis and haemostasis, 2025. 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.
- The Global Gap in the Hemophilia Paradigm Shift: Disparities in Research, Care, and Musculoskeletal Health.Hematology reports · 2026Review
- Hemophilia in the Era of Advanced Therapies: Structural Monitoring, the Role of Musculoskeletal Ultrasound, and a Proposed Multidisciplinary Care Model-A Structured Narrative Review.Diagnostics (Basel, Switzerland) · 2026Review
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Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
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.
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