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Efficacy and safety of hydroxyurea in children with E-beta thalassemia.

Subham Bhattacharya*, Habib Rahaman, Sumana Datta

Background: E-beta thalassemia is one of the commonest thalassemia syndromes in India. Regular blood transfusions became often the lifeline of the children, which causes serious morbidity and mortality. Hydroxyurea is expected to improve chronic anemia in these children, often at the cost of adverse effects. Data about the safety and efficacy of hydroxyurea in children with E-beta thalassemia is scarce.

Objectives: The present study was aimed to assess the efficacy and safety of hydroxyurea in reducing transfusion requirement in children with E-beta thalassemia, with monitoring of growth parameters. Methods: It was a prospective cohort study done in children with E-beta thalassemia, both Transfusions-Dependent (TDT) and Non-Transfusion-Dependent (NTDT), aged between 5-12 years. All children were treated with hydroxyurea with a dose of 10 mg/kg per day for 12 months, and followed up thereafter for at least 1 year.

Results and Conclusion: A total of 63 children were enrolled in the study. Hydroxyurea causes significant increment of baseline hemoglobin, both in TDT and NTDT. Transfusion requirement was significantly reduced in both the subgroups. There was no impairment of growth parameters during the study. Adverse effects were minimal and self-limiting. Hence the study concludes that, hydroxyurea is an effective and safe drug in children with E-beta thalassemia.