Opinion
Video
Real-World Insights: Factor vs Emicizumab in Hemophilia A
Steven W. Pipe, MD, discusses real-world evidence comparing factor replacement to emicizumab in hemophilia A, exploring efficacy, adherence, and cost considerations.
This is a video synopsis/summary of an Insights featuring Steven W. Pipe, MD.
Pipe delves into the real-world evidence surrounding factor replacement therapy and emicizumab in treating hemophilia A. With a robust clinical trial program involving hundreds of patients, the discussion extends to over 2300 individuals in real-world settings, shedding light on adherence, efficacy, and outcomes. The data suggests that patients on emicizumab prophylaxis consistently maintain low annualized bleed rates, often ranging from zero to 2, with a significant proportion achieving zero treated bleeds annually. Pipe explores switching studies, acknowledging mixed results, especially in regions with varying prophylaxis approaches. While annualized bleed rates may not always show a clear differentiator, patient preferences, quality of life measures, and activity levels strongly favor emicizumab due to its regimen ease, subcutaneous delivery, and steady-state levels. The interview also touches on cost considerations, noting that in the US, emicizumab's pricing might result in higher annualized costs compared to standard half-life agents. The comprehensive analysis underscores the importance of real-world evidence in understanding the practical implications and patient-centric benefits of these evolving hemophilia A therapies.
Video synopsis is AI-generated and reviewed by AJMC® editorial staff.
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