Modern approaches in hemophilia therapy

For 60 years the “gold standard” of hemophilia treatment has been the use of concentrates factors. In recent years, there has been a rapid development of therapeutics. Several extended half-life concentrates have been introduced into clinical practice, as well as a non – factorial type of therapy-a bispecific antibody mimicking activated factor VIII. Encouraging results in clinical trials show the use of antibodies to tissue factor pathway inhibitor, small interfering RNA to antithrombin and gene therapy. The advantages of new treatment methods are a reduction in the frequency of intravenous injections and improved adherence to therapy, especially in patients with inhibitors. Long-term safety and effectiveness require further study.

hemophilia, factor concentrates, emicizumab, gene therapy