New therapies for hemophilia

Abstract

Individuals with severe hemophilia have benefitted from 5 decades of advances that have led to widespread availability of safe and efficacious factors VIII and IX, a multidisciplinary integrated care model through a network of specialized hemophilia treatment centers, and aggressive introduction of prophylactic replacement therapy to prevent bleeding and preserve joint health. Yet, there are remaining challenges and treatment gaps which have prevented complete abrogation of all joint bleeding, and progressive joint deterioration may continue in some affected individuals over the course of a lifetime. Some of these challenges can now be addressed with recombinant clotting factors with extended half-life that may improve adherence to prophylaxis regimens through more convenient infusion schedules, maintain higher plasma levels for longer when clinically necessary, and allow for better adaptation to individual phenotypic and pharmacokinetic variability. Real-world case studies will be presented that illustrate practical application of these newly approved therapies in clinical practice and the clinical trial data that have demonstrated the potential for improved clinical outcomes by implementing these strategies.