Efficacy of immunomodulator drug thalidomide in multi-transfused thalassemia patients: A single center experience from South Punjab, Pakistan.

Abstract

Objective: To determine the efficacy of thalidomide in multi transfused Thalassemic Children. Study Design: Quasi-experimental study. Setting: Thalassemia Center, The Children’s Hospital and Institute of Child Health, Multan, Pakistan. Period: August 2023 to December 2024. Methods: A total of 121 transfusion dependent thalassemia (TDT) children of either gender, aged between 2 to 15 years, were analyzed. Non-probability, consecutive sampling technique was adopted. Children were given thalidomide at a dose of 1.5mg/kg/day. CBC evaluation and clinical examination were done at 3-months interval for a duration of 6 months. At each visit, side effects, subjective complaints and rise /fall of Hemoglobin was noted. Statistical analysis was conducted using IBM-SPSS version 26.0. Results: In a total of 121 children, the median age was 8.00 years (IQR, 5.00-11.00), while 77 (63.6%) children were males. At baseline, the median hemoglobin level was 8.00 g/dL (IQR, 6.75-8.75), increasing to 8.90 g/dL (IQR, 7.65-9.80) at 3 months, and 9.90 g/dL (IQR, 8.70-11.00) at 6 months (p<0.001). By the end of 6-months period, excellent, partial, and no response were noted among 59 (48.8%), 41 (33.9%), and 21 (17.4%) children, respectively. The most common treatment related side effects were constipation (n=9, 7.4%), neutropenia (n=6, 5.0%), and skin rash (n=5, 4.1%).No reported cases of severe adverse effects leading to discontinuation. Conclusion: In children with TDT, thalidomide significantly increased hemoglobin levels and reduced transfusion needs over six months.