Rebalancing Hemostasis

Overview

Therapeutic strategy for hemophilia that restores hemostatic balance by reducing anticoagulant activity rather than adding procoagulants; two main approaches are siRNA knockdown of antithrombin (fitusiran) and antibody inhibition of TFPI; both work across hemophilia A, B, and inhibitor patients. Rebalancing hemostasis approaches work by reducing the level of anticoagulants — either by knocking down antithrombin with fitusiran siRNA or by blocking TFPI with an antibody — rather than replacing the deficient procoagulant, and work independently of factor VIII or IX, covering all hemophilia types including inhibitor patients. Rebalancing hemostasis approaches require careful efficacy-safety calibration because reducing anticoagulants too aggressively risks thrombosis; the speaker noted that physicians must figure out how to use these new medicines, as both anti-TFPI and emicizumab combination events have resulted in patient deaths. (Rebalancing Approaches Introduced (15:00-20:00))