Treatment of haemophilia: From replacement to gene therapy

Abstract

Haemophilia A and B are congenital bleeding disorders characterized by missing or defective factor VIII or factor IX, respectively.

Factor replacement therapy has been the gold standard for prophylaxis and treatment of bleeding complications.

However, the inconvenience of regular intravenous administration, along with progression of arthropathy and development of inhibitors has driven the need for alternative treatment options, such as extended half-life products, non-factor coagulation products, such as subcutaneous emicizumab, blocking natural anticoagulants (rebalancing haemostatic agents) and gene therapy, which have been useful to control bleeding or are currently under late-phase clinical investigation.

CITATION  Med Clin (Barc). 2021 Sep 8;S0025-7753(21)00336-5. doi: 10.1016/j.medcli.2021.04.031