Long-term hydroxyurea treatment in young sickle cell patients.

Hydroxyurea is the first drug that, under well-organized, large-scale trials in adults, has shown a beneficial effect on the clinical course of sickle cell disease. Several small-scale trials have been conducted in children, but they used different therapeutic schedules, and only one was a single-blind crossover trial. Still, children are clearly good responders to the treatment because a rapid clinical improvement was observed, with decreased frequencies of vaso-occlusive crises, acute chest syndromes, and transfusion requirements. Despite large interindividual variations, virtually all the children studied increased their fetal hemoglobin, mean corpuscular volume, and total hemoglobin. Follow-up varied from 6 months to 59 months. More than in adults, the fetal hemoglobin increase was sustained, and few side effects were observed. Large-scale, placebo-controlled studies seem no longer needed. Guidelines concerning patient selection, dosing schedules, and monitoring protocols as well as exhaustive registries for the detection of long-term side effects are necessary.