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As newborns age, fetal hemoglobin is supplanted by adult hemoglobins (HbA, HbS, etc.), and infants with sickle cell disease become symptomatic. They experience pain crises, dactylitis, acute chest syndrome, and other complications of this disorder. Hydroxyurea (HU) is effective in decreasing sickle cell–related problems in adults and older children (JW Oncol Hematol May 6 2008), but is it safe and effective in infants?
To answer this question, pediatric investigators conducted a multicenter, randomized, double-blind, placebo-controlled trial of daily oral HU (20 mg/kg/day) in 193 infants (aged 9–18 months) with sickle cell anemia.
Those receiving HU versus placebo had lower incidence of painful events (177 vs. 375; P<0.001), dactylitis (24 vs…