Safety and Efficacy of Thalidomide Vs Hydroxyurea in β-Thalassemia patients: A Systematic Review of Current Evidence

Objective: The current systematic review was carried out to compare the safety and efficacy of thalidomide vs hydroxyurea in β-thalassemia patients. Methods: literature were extensively searched by using four databases: Web of Science, PubMed, Cochrane Library, and Scopus until January 22, 2024, for studies that did hit to hit comparison of thalidomide vs hydroxyurea based on safety and efficacy in β-thalassemia patients. Original research studies in English with observational and/or experimental designs, regardless of age and gender, used thalidomide for ≥3 months were included in this systematic review. Two independent reviewers extracted the data using a data extraction form. Results: Two studies collectively involving n=57 β-thalassemia patients fulfilled eligibility criteria. Thalidomide exhibited greater potential in improving Hb levels, HbF, and in reducing blood transfusion needs and serum ferritin levels compared to hydroxyurea in β-thalassemia patients. Mild adverse events (AEs) of non-serious nature were reported with both thalidomide and hydroxyurea. somnolence, and headache were the main AEs reported in patients treated with thalidomide, whereas abdominal pain, pruritis and gastritis were the main AEs reported in patients treated with hydroxyurea. Conclusion: This study demonstrates the higher potential of thalidomide in improving Hb levels and reducing transfusion requirements compared to hydroxyurea in β-thalassemia. However, these findings must be endorsed until well-designed clinical trials are conducted.