The current treatment of hemophilia relies on intravenous administration, repeated several times a week, of clotting factor VIII (FVIII) or factor IX (FIX) concentrates, either derived from plasma or produced by biotechnology. This preventive treatment is burdensome, expensive, and only allows for transient and partial corrections of the clotting factor deficiencies. Moreover, it is associated with the risk of developing neutralizing antibodies, termed inhibitors.
The FVIII and IX concentrates with a longer half-life, such as a bispecific monoclonal antibody mimicking the action of FVIII, as well as various strategies modifying the physiological regulation of coagulation, whilst being administered subcutaneously, represent new treatment options, either already validated or being evaluated. The first results of gene therapy studies also look very promising.
These developments offer patients with hemophilia new perspectives of treatment, if not cure, which this article proposes to review.