Abstract
Hemophilia is currently fully treatable by recombinant or plasma-derived factor VIII or IX concentrates. Coagulation factor concentrates are used to treat hemorrhages (on demand treatment) or for prophylactic treatment.
Common disadvantages of both groups of factor concentrates are their short half life and purely intravenous administration. Stronger or weaker immunogenicity of clotting factor concentrates may lead to inhibitor development which occurs in up to 30% of severe hemophiliacs and significantly complicates their treatment.
Current research focuses on development of prolonged half life clotting factor products, other than intravenous ways of their administration, and immunogenicity reduction. New concentrates for treatment of patients with inhibitors are also being developed which should have longer half life, better efficacy, and easier lab monitoring.
At the same time, intensive research in gene therapy continues at level of clinical trials already.