[Therapeutic innovations for hemophilia].Rev Med Suisse 2020; 16(676-7):47-52RM
The current treatment of haemophilia relies on intravenous administration several times a week of clotting factors VIII (FVIII) or IX (FIX) concentrates, either derived from plasma or produced by biotechnology. This preventive treatment is burdensome, expensive and associated with the risk of developing neutralizing antibodies (inhibitors). Factor VIII and IX concentrates with a longer half-life, a bispecific monoclonal antibody mimicking the action of FVIII as well as various strategies modifying the physiological control of coagulation and administered subcutaneously represent new treatment options, validated or being evaluated. The first results of gene therapy studies also seem very promising. These developments offer hemophiliacs patients new perspectives of treatment if not cure that this article proposes to review.