Hydroxyurea in Children with Beta Thalassemia Intermedia.

The beneficial effects of hydroxyurea (HU) have been well documented in sickle cell disease. Its usefulness in thalassemia syndromes has not been clearly elucidated. The aim of the study was to analyse the efficacy and side effects of HU in children with beta thalassemia intermedia (TI). The data of 73 children with TI were retrospectively analysed. Response to HU was assessed by change in hemoglobin (Hb) level and by a fall in transfusion requirements. An increase in Hb level was observed in 46 (63.0%) of 73 patients; the response was categorised as good in 43 and partial in 13 cases. There was a poor or no response in the remaining 27 (37.0%) children. Significant rise in Hb levels were documented after HU therapy; the responses were better in patients diagnosed beyond the age of 3 years (p=0.001). A fall in transfusion requirements by >50% was observed in 28 (66.7%) of the 42 patients who received transfusion prior to referral. There were no serious side-effects inspite of prolonged use. Transient neutropenia (<1.5x10⁹/L) was observed in 9 patients. A solitary case had severe myelotoxicity requiring discontinuation of HU. Over 60% of our patients showed good or partial response in terms of hemoglobin level and/or transfusion requirements. The study has drawbacks and limitations of retrospective analysis. The preliminary observations suggest the need for evaluating larger number of patients in a prospective manner of determine the optimum dose and duration of therapy.