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<0.001). 19 children (51.3%) had major response and 12 (32.4%) had moderate response. In 13.5% and 32.4% children response was observed within the first and second month of therapy, respectively. 15 (40.5%) children remained transfusion - free for a median (IQR) time of 6 (3-10) weeks of thalidomide therapy. Mean serum ferritin (SD) decreased from 1758.9 (835.1) to 1549.6(1016.9) (P<0.001). Mean HbF (SD) showed an increase from 2.95(2.6) to 49.2(33.3) (P<0.001). In 32 children, 47 adverse events were observed. Common adverse events were constipation and neutropenia (mostly mild).
Conclusions: Thalidomide resulted in major/moderate response in majority of children with transfusion-dependent thalassemia with satisfactory adverse effect profile.