Evolving Treatment Paradigms in Hemophilia

An overview of the biological and clinical distinctions between hemophilia A and B, including factor deficiencies, prevalence, diagnostic nuances, and implications for treatment strategy.

A historical perspective on traditional factor replacement therapies, tracing their development, successes, and ongoing limitations such as short half-life, infusion requirements, and inhibitor formation.

Exploration of the emergence of nonfactor treatment classes, explaining how these agents function independently of missing clotting factors and their relevance to patient convenience, dosing, and efficacy.

Detailed discussion of Fitusiran, Marstacimab, and Concizumab, their mechanisms of action, differentiation from factor replacement, and highlights from key clinical trials (BASIS, ATLAS, Explorer 8).

Focus on payer dynamics, cost, prior authorization processes, and the logistical aspects of transitioning patients from factor-based to nonfactor therapies, including adherence and at-home treatment implications.