Evolving Treatment Strategies in Hemophilia: Mechanisms, Benefits, and Practical Considerations

Hemophilia management has expanded beyond traditional factor replacement to include mimetics and rebalancing agents, each offering unique mechanisms and advantages. Factor concentrates—available as standard or extended half-life products and newer high-sustained/ULH versions—restore missing clotting factors directly, providing reliable control but requiring frequent infusions and monitoring for inhibitors. Mimetics, such as bispecific antibodies, bypass the need for factor VIII by bridging key steps in the coagulation cascade, offering more convenient dosing and reduced treatment burden. Rebalancing agents restore hemostatic balance by targeting natural anticoagulants, providing additional non–factor-based options. Their extended dosing intervals can ease clinic workload and enhance patient adherence, though they may require specialized monitoring such as antithrombin activity levels. Together, these therapies enable more individualized care while reducing bleeding risk and treatment complexity.