Impact of novel hemophilia therapies around the world.

ABSTRACT: Hemophilia A and B are hereditary bleeding disorders, characterized by factor VIII or IX deficiencies, respectively. For many decades, prophylaxis with coagulation factor concentrates (replacement therapy) was the standard-of-care approach in hemophilia. Since the 1950s, when prophylaxis started, factor concentrates have been improved with virus inactivation and molecule modification to extend its half-life. The past years have brought an intense revolution in hemophilia care, with the development of nonfactor therapy and gene therapy. Emicizumab is the first and only nonreplacement agent to be licensed for prophylaxis in people with hemophilia A, and real-world data show similar efficacy and safety from the pivotal studies. Other nonreplacement agents and gene therapy have ongoing studies

SUBMITTER: Ozelo MC 

PROVIDER: S-EPMC9004233 | biostudies-literature | 2022 Mar

REPOSITORIES: biostudies-literature