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Hemophilia in a Changing Treatment Landscape
Marie-Claude Pelland-Marcotte1, Manuel D Carcao2
1Division of Haematology/Oncology, Department of Paediatrics, The Hospital for Sick Children, University of Toronto, 555 University Avenue, Toronto M5G 1X8, Canada.
Abstract:
The mainstay of hemophilia management has been the regular, prophylactic infusion of missing coagulation factors VIII/IX. This approach is limited by the need for frequent intravenous infusions, high cost, limited availability, and the development of inhibitory antibodies to factors VIII/IX. Numerous recent breakthroughs are addressing many of these limitations. These include the development of extended half-life factors that require less frequent infusions and the development of various novel agents that can be given subcutaneously and infrequently, including FVIII-mimetic antibody and downregulators of natural anticoagulants. Finally, gene therapy is set to offer patients a possibility for a cure.
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