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Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective non-interventional study in a real-world setting

Haemophilia Feb 12, 2019

Kruse-Jarres R, et al. - Researchers examined adult/adolescent persons with haemophilia A (PwHA) without inhibitors, to evaluate annualized bleeding rate (ABR), haemophilia treatment practices and adverse events (AEs). Subjects aged ≥12 years with severe HA without history of inhibitors were considered eligible for inclusion in this study. Data for these participants were prospectively collected. This study included 94 participants (median [range] age, 34 [12-76] years), with a median (range) of 29.8 (12.4-47.7) weeks of monitoring. The reported treated bleeds in the episodic (n = 45) and prophylactic (n = 49) treatment groups were 872/1066 (81.8%) and 151/189 (79.9%), respectively; ABRs were 36.1 and 5.0, respectively, for treated bleeds and 43.1 and 6.2, respectively, for all bleeds. For these groups, respectively, 31.1 and 1.9 median (interquartile range) ABRs were reported for treated bleeds and 35.3 and 2.7 for all bleeds. Gastrointestinal polyp haemorrhage and haemarthrosis were documented as the serious AEs; the most common AE was viral upper respiratory tract infection. Overall, consistent with the literature, findings revealed continued bleeding on prophylaxis in PwHA without inhibitors and these patients needed treatment for breakthrough bleeds. Findings call for more efficacious haemostatic approaches.
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