ArticleResearch and practice in thrombosis and haemostasis2026
Outcomes of hemophilia A in children under primary and secondary prophylaxis: results from the HEMFIL Cohort Study.
Article in Research and practice in thrombosis and haemostasis, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Background: Early prophylaxis reduces the risk of joint damage in children with hemophilia A (CwHA). Long-term comparative data on bleeding outcomes and joint health in CwHA under primary prophylaxis (PP) and secondary prophylaxis (SP) remain limited. Methods: This prospective cohort study enrolled previously untreated or minimally treated (<5 exposure days) CwHA (factor VIII < 0.02 IU/mL). Patients were followed until 75 exposure days or inhibitor development. Annual bleeding rate (ABR) and annual joint bleeding rate (AjBR) were evaluated before and after prophylaxis initiation. Joint status was assessed once, at a median patient age of 8.4 years, using the Haemophilia Early Arthropathy Detection with Ultrasound and the Haemophilia Joint Health Score. Results: A total of 43 children were included (PP, Conclusion: In this study, CwHA receiving PP appeared to have a more severe bleeding phenotype compared with those on SP. However, this requires confirmation in future studies. Long-term joint outcomes were generally mild.
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