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RCTs demonstrate that non-clotting factor therapies reduce bleeds in hemophilia, highlighting treatment effectiveness.
Evidence from RCTs shows that prophylaxis using non-clotting factor therapies compared with on-demand treatment may reduce bleeding events, increase the percentage of individuals with zero bleeds, increase the incidence of non-serious adverse events, and improve HRQoL. Comparative assessments with other prophylaxis regimens, assessment of long-term joint outcomes, and assessment of economic outcomes will improve evidence-based decision-making for the use of these therapies in bleed prevention.
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Olasupo et al. (2026) studied this question.
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