EXPLORING NON-REPLACEMENT THERAPIES' IMPACT ON HEMOPHILIA AND OTHER RARE BLEEDING DISORDERS

The management of hemophilia, von Willebrand disease (VWD), and rare coagulation disorders traditionally relied on replacement therapies, such as factor concentrates, to address clotting factor deficiencies. However, in recent years, the emergence of non-replacement therapies has shown promise as an adjunctive approach, especially in hemophilia but also for patients with von Willebrand disease and rare bleeding disorders.This review article offers an overview of non-replacement therapies, such as FVIII-mimicking agents and drugs aimed at rebalancing hemostasis by inhibiting natural anticoagulants, particularly in the management of hemophilia. The utilization of non-replacement therapies in von Willebrand disease and rare bleeding disorders has recently attracted attention, as evidenced by presentations at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress. Non-replacement therapies provide alternative methods for preventing bleeding episodes and enhancing patients' quality of life, as many of them are administered subcutaneously and allow longer infusion intervals, resulting in improved quality of life and comfort for patients.