Follow-Up of Patients Undergoing Thalidomide Therapy for Transfusion-Dependent β-Thalassemia: A Single Thalassemia Center Cohort Study
DOI:
https://doi.org/10.36283/ziun-pjmd14-3/020Keywords:
β-Thalassemia, Hemoglobin, Thalidomide, Transfusion, ChelationAbstract
Background: Thalassemia is a common inherited disorder globally. This study aimed to evaluate the long-term effects of thalidomide therapy in patients with transfusion-dependent β-Thalassemia (TDT).
Methods: This retrospective cohort study was done at the Thalassemia Center of Dera Ghazi Khan Teaching Hospital, Pakistan, from October 2019 to September 2024. A total of 500 children aged 1-15 years, diagnosed with TDT, and who had received thalidomide were analyzed. Non-probability, consecutive sampling technique was adopted. Necessary laboratory and clinical parameters were evaluated during the 5-year follow-up period. Data analysis was performed applying IBM-SPSS Statistics, version 26.0. Chi-square or Fisher’s exact test was used to compare qualitative data, while ANOVA or independent sample t-tests were performed for quantitative data, considering p<0.05 as significant.
Results: Out of a total of 500 patients, 256 (51.2%) were female. The mean age was 8.25±2.19 years. The mean baseline Hb was 6.5±0.6 g/dl, rising to 8.5±0.5 g/dl at 6 months, and stabilizing at 8.6±0.4 g/dl at 1 year and 8.6±0.3 g/dl at 5 years (p<0.001). At baseline, all required monthly transfusions, with 307 (61.4%) becoming transfusion-independent by 6 months, 383 (76.6%) by 1 year, and 410 (82%) by 5 years (p<0.001). Chelation therapy was required for 453 (90.6%) patients at baseline, 308 (61.8%) at 6 months, 346 (69.2%) at 1 year, and 57 (11.4%) by 5 years (p<0.001). Significant liver, and spleen regression were noted by the end of the study period (p<0.001). All patients experienced abdominal distension, which resolved within 1 year, while 2 (0.4%) patients had cerebrovascular accidents.
Conclusion: Thalidomide therapy significantly improved hemoglobin levels, reduced transfusion and chelation needs, and led to organ size regression over five years in children with TDT, with minimal adverse effects, supporting its long-term efficacy and safety.
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