Therapeutic innovations for hemophilia

Authors: Hermans, C. and Lambert, C.

Rev Med.Suisse; 16,676-7:47-52. January 2020

Affiliations: Unité d’hémostase-thrombose, Service d’hématologie, Cliniques universitaires Saint-Luc, Université catholique de Louvain, Bruxelles.

Abstract: 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 hemophiliac patients new perspectives of treatment if not cure that this article proposes to review.