Enabling normal psychophysical development in children with hemophilia: the choice for prophylaxis

Abstract

Hemophilia A and B, the congenital deficiencies of coagulation factors VIII and IX, are characterized by recurrent joint and muscle bleeding episodes and progressive musculoskeletal damage (hemophilic arthropathy). Primary prophylaxis – that is, the regular infusion of factor concentrates after the first hemarthrosis and/or before 2 years of age – is now recognized as the first-choice treatment for children with severe hemophilia. Preventing bleeding from an early age enables avoidance of the clinical impact of hemophilic arthropathy and the consequences regarding psychosocial development and quality of life for these children. Interestingly, recent data suggest a role for early prophylaxis in also preventing inhibitor development, the most serious complication of treatment in hemophilia. Secondary prophylaxis, initiated after 2 years of age or after two or more joint bleeds, aims to avoid (or delay) the progression of arthropathy. In addition, better outcomes and better quality of life have been reported with earlier treatment. This review summarizes the evidence, current clinical strategies and open issues regarding prophylxis in children with hemophilia.