Sickle Cell Anemia Toddlers Benefit from Maximal Hydroxyurea Doses, Study Finds

Intensifying treatment with hydroxyurea in young children with sickle cell anemia is safe and improves several disease parameters in the long run, researchers demonstrated in a clinical trial (NCT00305175) comparing toddlers with older children.

Based on the positive findings, researchers argue that the practice of giving small children higher doses of the drug needs to be further studied. A new clinical trial (NCT03020615) addressing these matters is in the planning stage.

The study, “Hydroxyurea at Maximal Tolerated Dose (MTD) Prior to Completion of the β-Globin Switch Has Additive but Not Sustained Benefits in Fetal Hemoglobin Production,” was presented at the ASH 2016 Annual Meeting.