Efficacy of hydroxyurea in Thalassemia major patients

Abstract

Background: Purpose of this study was to determine the efficacy of hydroxyurea in Thalassemia major patients. Non-randomized, interventional, prospective single centre study. Methods: Hydroxyurea (HU) was administered orally to Beta-thalassemia major patients in doses of 8-15mg/kg/day. They were followed up for a period of 6 months. Before starting hydroxyurea, all patients underwent routine biochemical laboratory tests. Response was evaluated by observing the children for a rise in haemoglobin and Hb F levels and decreasing serum ferritin and transfusion requirement. Pre-HU and post-HU groups were made to compare the parameters after HU therapy. Treatment was stopped transiently if features of hepato-toxicity and myelo-toxicity appear. Clinically, the response was categorized as - good if mean haemoglobin increased >2 g/dl, partial when haemoglobin increased by 12 g/dl and the rest were categorized into non- responder group. Results: Total 60 patients were recruited with age range of 2-16 years (mean age 8.6 years) and the mean age of presentation with anaemia was 1.68 years. It was observed that 13/60 (21.66%) had a good response, 18/60 (30%) had a partial response and 23/60 (38.34%) had no response while 6/60 (10%) could not be evaluated, as they dropped out. The mean spleen size decreased significantly (P<0.001), mean monthly transfusion volume decreased significantly (P<0.02), mean Hb level increased significantly (P<0.001), mean Hb-F level increased significantly (P<0.05) and mean serum ferritin level decreased significantly (P<0.01) in good responders. Better response was seen with higher dosage regime of the drug. Conclusion: Hydroxyurea has a definite role in the overall management of patients with Thalassemia Major to decrease the need for regular transfusions and concomitant iron load.