Long-term aspects of hemophilia B treatment: part II.

This paper focuses on the benefits of prophylaxis, compared with on-demand therapy, in achieving long-term treatment goals in hemophilia patients. While primary treatment goals are to prevent joint damage and improve quality of life, other issues such as treatment costs-balanced against improvements in health status and direct and indirect costs of hemophilic arthropathy-need to be considered. The ESPRIT study (Evaluation Study on Prophylaxis: a Randomized Italian Trial), a 10-year trial comparing on-demand and prophylactic treatment in terms of efficacy, safety, and cost, is discussed. Early results support findings from other studies regarding the benefits of prophylaxis. Prospective pharmacoeconomic evaluations are needed to help define optimal treatment programs for patients with hemophilia A and B.