Limitations of prophylactic treatment in patients with hemophilia

Abstract

: Prophylaxis with factor VIII concentrates is the cornerstone of treatment for severe hemophilia A. In children, early onset of primary and secondary prophylaxis is the gold standard in most of countries with adequate financial resources. In adults, it is reasonable to continue the prophylactic treatment initiated during childhood to preserve joint functionality. Adults with advanced hemophilic arthropathy can also benefit from tertiary prophylaxis, showing reductions in bleeding episodes, target joints and missed days from work. The long-term evolution of hemophilic patients usually depends on how successfully prophylaxis can prevent bleeding in both children and adults. For prophylaxis to be efficient it is essential to consider the resources available (factor concentrate use and trough levels) and the patient's bleeding phenotype and clinical situation (levels of activity required, presence of chronic synovitis and arthropathy), and to define the annual number of bleeds (particularly into joints) acceptable for each patient.