Gideon Koren1,2*, Michael Lishner3,4, Ami Ballin4
Hydroxyurea (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 doses had to be fluctuated daily to achieve the aimed mean dose. Because adherence improves with unchanged daily dose, and due to the more than 10 fold variability in HU pharmacokinetics in children, there was an urgent need for a pediatric formulation of HU.
This issue has been solved with 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.
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