Understanding Hemophilia A and B

The MHE Hemophilia K-Cast Series provides a comprehensive exploration of how hemophilia treatment has evolved over time, highlighting both scientific breakthroughs and healthcare delivery implications. It begins with a clear differentiation between hemophilia A and B—covering diagnosis, factor deficiency, symptoms, and prevalence—before tracing the history of factor replacement therapies that have long been the standard of care. Dr. Annette Von Drygalski outlines how these factor-based treatments improved outcomes yet left persistent challenges such as frequent intravenous infusions, inhibitor development, and inconsistent bleed control

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The series then transitions to a deep examination of the new generation of nonfactor therapies—notably Fitusiran, Marstacimab, and Concizumab—that are redefining hemophilia management. Viewers learn about the mechanisms of action for these agents, including rebalancing the coagulation system through targeting natural anticoagulants or other novel pathways. The discussion explores how these therapies differ from factor replacement in dosing, route of administration (often subcutaneous), and real-world practicality. Dr. Von Drygalski references pivotal clinical trials such as BASIS, ATLAS, and Explorer 8 that demonstrate efficacy and safety, setting a foundation for their integration into clinical practice

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Finally, the series addresses the real-world implications of these innovations. It discusses payer and access considerations, including prior authorization hurdles and cost management, while emphasizing how nonfactor therapies could reshape patient experiences by reducing treatment burden and allowing at-home administration. The series closes by reflecting on how the patient-clinician dynamic may shift toward more collaborative, adherence-focused care models and what these therapeutic advancements mean for the broader managed care community