SAFETY AND EFFICACY OF THALIDOMIDE IN TRANSFUSION-DEPENDENT β-THALASSEMIA: A SYSTEMATIC REVIEW AND META-ANALYSIS

Abstract

OBJECTIVE: The current meta-analysis was carried out to identify the efficacy and safety of thalidomide in transfusion-dependent β-thalassemia (TDT) patients. METHODS: Six databases: PubMed, EMBASE, Scopus, Cochrane Library, EBSCOhost, and MEDLINE were searched until November 18, 2021, for studies that assessed the efficacy of thalidomide in TDT patients by using the following search terms: “Thalidomide”, “thalidomid”, “thalomid”, “N-phthaloylglutamimide”, and Thalassemia” using Boolean or wildcard operators. Original research publications in English with observational and/or experimental designs having a sample size ≥10, regardless of age and gender, used thalidomide for ≥3 months exploring the impact of thalidomide in ameliorating transfusion needs among TDT patients were included in this meta-analysis. Data were independently extracted by two reviewers using a data extraction form. The National Institutes of Health tool was used for quality assessment. RESULTS: Nine studies collectively involving 407 TDT patients fulfilled eligibility criteria. Thalidomide was associated with complete cessation of blood transfusion requirements with an overall response of 54% (95% CI, 34–75%) to a transfusion-independent state; heterogeneity was considered high with an I2 of 94.7%, p-value<0.001. Mild adverse events were reported in 44% of patients. CONCLUSION: Thalidomide is a well-tolerated, effective and safe drug among TDT patients, these findings, however, should be confirmed through well-designed clinical trials. PROSPERO Review registration number: CRD42021289950.

Keywords

• thalidomide
• safety
• efficacy
• transfusion-dependent β-thalassemia
• thalassemia
• beta-thalassemia
• hemolytic anemia
• congenital hemolytic anemia