The Fourth Annual Meeting of the International Network for Pediatric Hemophilia: Current Challenges and Recommendations in the Clinical Care of Children with Hemophilia.

The International Network for Pediatric Hemophilia (INPH) comprises a group of physicians committed to the unique care of and challenges facing pediatric hemophilia patients. By collaborating on an international level, extensive experience can be shared on current practice, new trends can be discussed and scientifically valid studies can be developed and performed. The three overall objectives of the group (scientific progress, education and networking) are achieved at each annual meeting starting with a round table on the members' current research and clinical activities, project reports of INPH study initiatives, followed by invited educational presentations and interactive discussions. The meetings close with proposals of new projects, future directions of the group and concluding remarks. The Fourth Annual INPH meeting, held in 2009 in Boston, MA, USA, focused on inhibitor development and hemophilic arthropathy in the clinical care of children with hemophilia.

Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia.

BACKGROUND: Effective ways to prevent arthropathy in severe hemophilia are unknown. METHODS: We randomly assigned young boys with severe hemophilia A to regular infusions of recombinant factor VIII (prophylaxis) or to an enhanced episodic infusion schedule of at least three doses totaling a minimum of 80 IU of factor VIII per kilogram of body weight at the time of a joint hemorrhage. The primary outcome was the incidence of bone or cartilage damage as detected in index joints (ankles, knees, and elbows) by radiography or magnetic resonance imaging (MRI). RESULTS: Sixty-five boys younger than 30 months of age were randomly assigned to prophylaxis (32 boys) or enhanced episodic therapy (33 boys). 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). High titers of inhibitors of factor VIII developed in two boys who received prophylaxis; three boys in the episodic-therapy group had a life-threatening hemorrhage. Hospitalizations and infections associated with central-catheter placement did not differ significantly between the two groups. CONCLUSIONS: Prophylaxis with recombinant factor VIII can prevent joint damage and decrease the frequency of joint and other hemorrhages in young boys with severe hemophilia A. (ClinicalTrials.gov number, NCT00207597 [ClinicalTrials.gov].).