Efficacy and Safety of Thalidomide in Patients With Transfusion-Dependent Thalassemia

Objective To assess the efficacy and safety of thalidomide in children with transfusion-dependent thalassemia. Methods This prospective, single center, open-label study enrolled children aged 12–18 years, and who received thalidomide for a duration of 6 months at a starting dose of 2–3 mg/kg/day. Efficacy was assessed by reduction in transfusion requirement and rate of fall of hemoglobin. Efficacy was classified as major, moderate and minimal/no response depending on the reduction in transfusion requirement. Safety was assessed by adverse effects related to thalidomide. Results 37 children [mean (SD) age, 14.7 (1.8) years were included. Rate of fall of hemoglobin reduced from a mean of 1.0 (0.24) g/week pre-thalidomide therapy to 0.58 (0.26) g/week after 6 months of thalidomide ( P