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.

Process of Normalization in Families With Children Affected by Hemophilia

“…Consequently, in this article we focus on (a) common themes in conceptions of family life and chronic hemorrhagic disease held by families who share the condition of having a hemophiliac child; and (b) how the various therapeutic regimes (on-demand or prophylaxis) affect the different modalities and levels of normalization. With regard to this last point, there are indeed scholars (Coppola et al, 2010) who have claimed that prophylaxis normalizes family life all on its own, because it reduces parents’ anxieties concerning emergencies and allows children to play with other children in a normal way. They have also pointed out that because infusions are regular, families find it easier to sustain their regular, everyday routine.…”