Update on hemostatic treatment for hemophilia patients

Abstract

The supplementation therapy with factor (F)VIII or FIX products for hemophilia patients has resulted in the prevention of the progression of arthropathy and the great improvement of their quality of life (QOL). There have been serious issues, however, associated with this therapy, such as the requirement of frequent intravenous infusion, anti-FVIII or FIX alloantibody (inhibitor) development, and difficulty of hemostatic treatment for patients with inhibitor development. To solve these unmet needs, some extended-half-life FVIII or FIX products and non-clotting factor products have been developed. An anti-FIX/FX bispecific antibody, emicizumab, having the features of long half-life and subcutaneous administration, has resulted in the marked prevention of bleeding in severe hemophilia A patients, irrespective of the presence of the inhibitor. Furthermore, clinical trials using novel products that suppress the anti-coagulation function, which are based on the concept of rebalancing coagulation, are now ongoing. These new therapeutic approaches for hemophilia, which shift the paradigm of hemophilia treatment, could provide further improvement of the QOL of pediatric patients.