A Randomized Clinical Trial of Prophylaxis in Children with Hemophilia A (the ESPRIT Study).
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Background: Prevention of arthropathy is a major goal of hemophilia treatment. While studies in adults have demonstrated an impact of prophylaxis on the incidence of joint bleeds and patients' well-being in terms of improved quality of life (QoL), it is unclear whether or not prophylaxis influences the outcome and well-being perception of children with hemophilia. Objective: This randomized controlled study compared the efficacy of prophylaxis with episodic therapy in preventing hemarthroses and image-proven joint damage in children with severe hemophilia A (factor VIII <1%) over a 10-year time period. Methods: Forty-five children with severe hemophilia A, aged 1-7 years (median 4), with negative clinical-radiological joint score at entry and at least one bleed during the previous 6 months, were consecutively randomized to prophylaxis with recombinant factor VIII (25 IU/Kg 3x week) or episodic therapy with ≥25 IU/Kg every 12-24 hours until complete clinical bleeding resolution. Safety, feasibility, direct costs and QoL were also evaluated. Results: Twenty-one children were assigned to prophylaxis, 19 to episodic treatment. Children on prophylaxis had fewer hemarthroses than children on episodic therapy: 0.20 vs. 0.52 events/patient/month (p<0.02). Plain-film radiology showed signs of arthropathy in 6 patients on prophylaxis (29%) vs. 14 on episodic treatment (74%) (p<0.05). Prophylaxis was more effective when started early (≤36 months) with patients having less joint bleeds (0.12 joint bleeds/patient/month) and no radiologic signs of arthropathy. Conclusion: 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.