After decades of a single approach—replacement factor infusion—the treatment landscape for the management of hemophilia is rapidly evolving and numerous exciting research avenues are being pursued. The WFH Novel Therapies eLearning Centre brings together introductory information and in-depth resources on these new developments in a convenient online hub that is updated often.
Hemostatic balance is a fine balance between too much clotting (thrombosis) and not enough clotting (bleeding). In hemophilia, an essential clotting factor is lacking, and the hemostatic balance is tipped toward too much bleeding. The current standard of care for hemophilia is life-long prophylaxis with clotting factor concentrates to replace the clotting factor that is missing and to prevent spontaneous bleeds. However, access to these products is restricted in many contexts. Furthermore, factor replacement products have several limitations, including the characteristic saw-tooth rise and fall in factor levels observed after each infusion. In the absence of stable factor levels, the body has reduced ability to form clots and to prevent and stop bleeds (hemostasis), which can lead to bleeding events and joint damage between infusions.