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About 300f children with classical hemophilia (factor VIII [FVIII] deficiency) develop alloantibodies (inhibitors) when exposed to FVIII concentrates. These antibodies inactivate FVIII, greatly complicating the treatment of this bleeding disorder. Thus, inducing tolerance to FVIII early in childhood is a mainstay of contemporary hemophilia management.
To determine the optimal regimen for tolerance induction, an international consortium conducted a randomized comparison of high-dose FVIII (HD, 200 IU/kg daily) versus low-dose FVIII (LD, 50 IU/kg 3 times/week) in 115 children (median age, 15.5 months). Inhibitor titers were measured every 4 weeks, and, when the inhibitor became undetectable, FVIII recovery and half-life were determined. Succes…