Prophylactic infusion regimens in the management of hemophilia

Abstract

To summarize, prophylactic treatment of hemophilia begun at an early age has been gaining acceptance as the optimal therapeutic option in an increasing number of hemophilia centers in the developed world in recent years. In all too many parts of the world, however, this option must be viewed as a long-range goal in hemophilia care, since national economic resources are insufficient for regular prophylactic treatment to be feasible at the moment. The future development of prophylaxis seems to be focused on three different areas. First, research has focused on improving the cost-effectiveness of the current model by testing daily, frequent injections using novel devices for venous access or continuous infusion with portable or implantable mini-pumps and administration of bio-engineered concentrates with a prolonged half-life at a reduced price. Secondly, a break-through in gene therapy, which will enable us to introduce a gene producing an amount of clotting factor sufficient to provide a continuous prophylactic concentration in the blood. Finally, the most mind-challenging option makes most of the discussion in this chapter obsolete and focuses on the development of an oral compound, peptide, or peptidomimetic agent with the capacity to activate the coagulation cascade in a controllable way.