New therapies for hemophilia.

摘要: Hemophilia A (HA) and hemophilia B (HB) are the most common severe bleeding disorders. Replacement therapy, providing missing coagulation factor, has been mainstay of treatment both prophylactically to treat bleeding. Despite widespread availability safe effective replacement patients with HA HB continue experience a tremendous burden treatment, breakthrough bleeding, progressive joint disease, as well high rates inhibitor development. These remaining challenges now being addressed by incredible advances in bioengineering. Recombinant bioengineering led therapies easier modes administration, decreased immunogenicity, increased efficacy, extended half-lives. Emicizumab, bispecific antibody that acts substitutive therapy for HA, approved without inhibitors. Novel compounds development exploit natural balance hemostasis targeting anticoagulants protein C, S, tissue factor pathway inhibitor, antithrombin. The substitution rebalancing provide an opportunity steady-state hemostatic control exposure immunogenic clotting proteins. As such, they may have broader applications outside those investigated clinical trial programs.