Safety and Effectiveness of Thalidomide in Transfusion-Dependent and Non-Transfusion-Dependent Thalassaemia Children in Eastern India.

Thalassaemia is a major public health concern in Eastern India, with many children requiring lifelong blood transfusions. Thalidomide, known for its immunomodulatory properties, has shown promise in reducing transfusion needs by increasing fetal hemoglobin production.The safety and efficacy of thalidomide was evaluated in 404 patients aged 2 to 18 years, diagnosed with beta thalassaemia major or intermedia.Mean age was 8.4 years with 54.5 % cases being male. Majority of the patients were Beta thalassaemia major (54.8%). Mean Haemoglobin ± SE at baseline was 6.435 ± 0.092, with a significant increase in haemoglobin levels from 6.435 ± 0.092 at baseline to 8.157 ± 0.069 at the 12 months (p < 0.001). The mean number of transfusions decreased from 1.261 ± 0.051 at 3 months to 1.108 ± 0.074 at 12 months (p < 0.001). The mean spleen size decreased from 3.712 ± 0.087 at baseline to 2.242 ± 0.068 at 12 months (p < 0.001). The mean ferritin level decreased from 1947 ng/mL at baseline to 1554 ng/mL at 12 months (p < 0.001). This decline in ferritin reflects both reduced transfusion requirements and improvement in iron overload. Total bilirubin levels also showed a significant downward trend over 12 months (p < 0.001). At the end of 12 months, 30.7% of the responses were excellent, 52.5% were good, 13.3% were partial and rest 3.5% were no response. Side effects were reported in 62.6% of the patients, out of which most common was constipation (51%).Thalidomide significantly benefits transfusion-dependent thalassaemia patients, with a high proportion showing excellent or good response.