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In two randomized trials, researchers studied options for preventing adverse events in children with sickle cell disease (SCD). One focused on hydroxycarbamide (hydroxyurea [HU]), the other on the antiplatelet drug prasugrel.
In a U.S. study, 121 children (aged 4–16 years) with elevated transcranial Doppler (TCD) flow velocities (≥200 cm/sec) but no severe vasculopathy were randomly assigned to continue standard monthly transfusion therapy (hemoglobin S target, ≤30%) or switch to the maximally tolerated dose of HU. With respect to TCD velocity, HU was statistically noninferior to chronic transfusion (mean, 138 cm/sec vs. 143 cm/sec). No participants had a stroke; three transient ischemic attacks occurred in each group. Although HU recipients…