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Prophylaxis with recombinant factor VIII can prevent joint damage and decrease the frequency of joint and other haemorrhages in young boys with severe haemophilia A, a small study has concluded.
In the study, 65 five boys younger than 30 months of age with severe haemophilia A were randomly assigned to regular infusions of recombinant factor VIII (prophylaxis, n=32) or to an enhanced episodic infusion schedule of at least three doses totalling a minimum of 80IU/kg bodyweight of factor at the time of a joint haemorrhage (n=33). When the boys reached six 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, 1.5–24.4). The mean annual numbers of joint and total haemorrhages were higher at study exit in the episodic-therapy group than in the prophylaxis group (p<0.001 for both comparisons).
High titres of inhibitors of factor VIII developed in two boys who received prophylaxis; three boys in the episodic-therapy group had a life-threatening haemorrhage. Hospitalisations and infections associated with central-catheter placement did not differ significantly between the two groups.
N Engl J Med 2007;357:535-44,603-5