The Case for Prophylaxis in Children

• In the Joint Outcomes Study 65 boys aged >30 months were randomly assigned to prophylaxis (n=32) or enhanced episodic therapy (n=33) with rFVIII
• When the boys reached 6 years of age, 93% of those in the prophylaxis group and 55% of those in the episodic-therapy group were considered to have normal index-joint structure on MRI (P=0.006)
• The relative risk of MRI-detected joint damage with episodic therapy as compared with prophylaxis was 6.1 (95% confidence interval, 1.5 to 24.4)
• The mean annual numbers of joint and total hemorrhages were higher at study exit in the episodic-therapy group than in the prophylaxis group (P<0.001 for both comparisons)

 

Mark T. Reding, MD Associate Professor of Medicine Director, Center for Bleeding and Clotting Disorders University of Minnesota Medical Center