BackgroundHydroxyurea (HU) is the only FDA- approved disease- modifying drug for sickle cell disease, by inducing the production of fetal hemoglobin and thus decreasing the sickling of red blood cells. Till recently HU was available only in adult doses of 1000 mg. This meant that to aim at the standard dose of 20 mg/kg/d, most young children had to be overdosed, or the dose had to be fluctuated daily to achieve the aimed mean dose. Because adherence improves with unchanged daily dose, and due

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... the more than 10 fold variability in HU pharmacokinetics in children, there was an urgent need for a pediatric formulation of HU.Methods and resultsThis issue has been solved with the FDA approval of the French-originated orphan HU, Siklos, a preparation of 50 and 100 mg, which prevents the risk of inappropriate dosing in children.Studies show that the child appropriate dose preparation much more closely allow young children to receive appropriate dose of Hydroxyurea, and increase adherence with this critical drug.ConclusionsIn summary, FDA approval of the French-originated orphan HU, Siklos in preparations of 50, 100 mg, prevents the risk of inappropriate dosing of the drug in children. This should encourage all involved in pediatric medicine, from health care physicians and pharmacists, to the pharmaceutical industry and regulators to act similarly in other therapeutic areas where inappropriate pediatric dose schedules are endangering the health and wellbeing of children.Disclosure(s)G Koren has been a consultant for Medunik USA.