TL;DR
Current and emerging non-factor therapies for hemophilia are summarized, focusing on their mechanisms of action, clinical evidence, safety considerations, unmet needs, and future perspectives for safe implementation in clinical practice.
While conventional prophylaxis with clotting factor concentrates (CFC) is the standard of care for hemophilia A and B, its challenges include frequent intravenous administration, factor level fluctuations, and potential inhibitor development. Rebalancing agents, including fitusiran (antithrombin-lowering small interfer...
INTRODUCTION Hemophilia A is an inherited bleeding disorder due to factor VIII (FVIII) deficiency and is characterized by recurrent bleeding and progressive joint damage. For the last two decades, treatment strategies have expanded from FVIII replacement therapy to include extended half-life products, non-factor therap...
The therapeutic landscape of hemophilia, a rare inherited bleeding disorder, has rapidly evolved, introducing several novel modalities: extended half-life (EHL) coagulation factor concentrates, non-factor therapies, hemostasis-rebalancing agents, and gene therapy. These advances have improved clinical outcomes by reduc...
ABSTRACT Introduction Hemophilia A (HA) management has progressed significantly, yet standard and extended half-life (EHL) clotting factors present persistent limitations, including intravenous burden and suboptimal trough levels. Areas covered This narrative review highlights recently approved agents and phase III can...
A pattern emerges, where FXI/FXIa inhibition offers a favorable safety profile across multiple settings and high-risk subgroups, yet clear efficacy has so far been established only in secondary prevention of noncardioembolic ischemic stroke over existing antiplatelet strategies.
Hemophilia is a rare X-linked inherited bleeding disorder caused by a deficiency of coagulation factor VIII (hemophilia A) / factor IX (hemophilia B), resulting in impaired clot formation and recurrent bleeding episodes. This review aims to provide a comprehensive overview of the epidemiology, pathophysiology, diag...
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