Hemophilia in the practice of a general practitioner and hematologist

Hemophilia is a rare disease whose approved treatment options have remained virtually unchanged for 50 years. However, over the past decade there has been an innovation boom in treatment options that are either under development or have been approved for the treatment of hemophilia, including engineered clotting factors and a vast range of new approaches and techniques. Some of these new techniques, especially gene therapy, show proof of principle in hemophilia, but may have wider applications. These advances, combined with improved diagnostics, are now enabling clinicians to improve standards of care for people with hemophilia. The various mechanisms of action and modifications used in these therapies have implications for their safe and effective use, which must be balanced with their therapeutic utility.