Prophylaxis Prevents Bleeds and Arthropathy in Children with Hemophilia A (the ESPRIT Study)

A multinational European research team has reported findings from a randomized controlled trial that compared the efficacy of prophylaxis with episodic (on-demand) therapy in children with severe hemophilia over a 10-year time period. The objective of this study was to learn whether a prophylaxis strategy reduces the incidence of joint bleeds (hemarthroses) and image-proven joint damage.

Forty-five children with severe hemophilia A, aged 1 to 7 years with negative clinical-radiologic joint score at entry, were consecutively randomized to prophylaxis with recombinant factor VIII (25 IU/kg three times weekly) or on-demand therapy with ≥25 IU/kg every 12 to 24 hours until complete resolution of clinical bleeding. Ultimately, 21 children were assigned to prophylaxis and 19 to on-demand treatment. Children on prophylaxis had fewer joint bleeds than children on on-demand therapy: 0.20 vs. 0.52 events per patient per month (P<0.02). Plain-film radiology showed signs of arthropathy in 29 percent of patients on prophylaxis vs. 74 percent receiving on-demand treatment (P<0.05).

Prophylaxis was more effective when started before 36 months of age; that subset of patients experienced fewer joint bleeds (0.12 joint bleeds per month) and no radiologic signs of arthropathy. This randomized trial confirms the efficacy of prophylaxis in preventing bleeds and arthropathy in children with hemophilia, particularly when it is initiated early in life.