Long term thalidomide therapy's efficacy and safety in transfusion dependent beta thalassemia major patients: a systematic review

OBJECTIVE: To identify the effectiveness and safety of thalidomide in transfusion-dependent beta Thalassemia Major (TDBTM) patients. METHODS: A comprehensive search of PubMed, Cochrane Library, and Embase was conducted between January 01, 2014, and April 17, 2024, using terms like "thalidomide", "thalidomide", "alpha- phthalimidoglutarimide", "thalassemia", "beta thalassemia", "transfusion dependent thalassemia" using Boolean or wildcard operators. Studies published in English with an observational or experimental design, including more than 10 TDBTM patients treated with thalidomide for at least 3 months, were included. The review focused on patients of all ages and genders, evaluating the impact of thalidomide on transfusion requirements. The included trials, involved 780 participants (age range=1.5-27.2 years), showing improvements in hemoglobin, fetal hemoglobin (HbF), serum ferritin, spleen size, and quality of life. National Institute of Health tool was used for quality assessment. RESULTS: After screening 19462 records, 18147 remained after duplicates were removed. Of these, 18138 were excluded, leaving 09 studies for inclusion in the review. Conducted in Pakistan, China, India and Iraq (2014-2024), the studies included a single-arm trial, a double-blinded RCT, and an open-label RCT and Pre-post enrolling 780 participants. Thalidomide (50-150 mg/day) improved hemoglobin, HbF, serum ferritin, spleen size, and quality of life, with a mean follow-up of 13 months. Thalidomide therapy resulted in transfusion independence in 69.6 % (n= 543) patients. Adverse effects were reported in 41.2 % (n=322) patients. Studyqualitywas rated as good. CONCLUSION: Thalidomide is a well-tolerated, safe, and effective treatment for TDBTM patients, but these findings require confirmation through well- designed clinical trials.