Abstract
At present, the main therapies for ß-thalassemia patients include regular blood transfusion and iron chelation, associating with a number of limitations. Thalidomide, a fetal hemoglobin (HbF) inducer that promotes γ-globin gene expression, has been reported to be effective for ß-thalassemia. Thus, this meta-analysis was conducted to assess the efficacy and safety of thalidomide for treating patients with ß-thalassemia. We searched the related studies from eight databases published from inception until December 1, 2021. The R 4.0.5 language programming was used to perform meta-analysis. After screening of retrieved articles, 12 articles were included that enrolled a total of 451 patients. The Cochrane Collaboration risk assessment tool was used to evaluate the quality and the bias risk of the randomized controlled trials (RCTs), and non randomized trials were assessed using Newcastle-Ottawa Scale (NOS). After treatment with thalidomide, the pooled overall response rate (ORR) was 85% (95% confidence interval (CI): 80–90%), and the pooled complete response rate (CRR) was 54% (95% confidence interval: 31–76%). Compared with the placebo group, the thalidomide group had higher odds of overall response rate (odds ratio = 20.4; 95% CI: 6.75–61.64) and complete response rate (odds ratio = 20.4; 95% CI: 6.75–61.64). A statistically significant increase in hemoglobin level and HbF level after treatment, while there was no statistically significant difference in adult hemoglobin (HbA) level, spleen size, and serum ferritin. According to the results of ORR and CRR, transfusion-dependent thalassemia (TDT) patients showed remarkable efficacy of thalidomide, 83 and 52% respectively. So we analyzed 30 transfusion-dependent thalassemia patients from three studies and found that the most frequent ß-globin gene mutations were CD41-42 (-TCTT), while response to thalidomide did not show any statistically significant relationship with XmnI polymorphism or CD41-42 (-TCTT) mutation. About 30% of patients experienced mild adverse effects of thalidomide. Collectively, thalidomide is a relatively safe and effective therapy to reduce the blood transfusion requirements and to increase Hb level in patients with ß-thalassemia.
Highlights
Β-thalassemia comprises a group of hereditary hematological disorders characterized by abnormalities in the synthesis of the ß chains of hemoglobin
Jain et al conducted a randomized controlled trials (RCTs) and compared hydroxyurea arm with thalidomide arm, while only thalidomide arm was included in the present metaanalysis (Jain et al, 2019)
One RCT study and 11 singlearm studies (4 retrospective and eight prospective studies) were included, of which 6 studies were conducted in China, three in India, two in Bangladesh, and one in Iraq
Summary
Β-thalassemia comprises a group of hereditary hematological disorders characterized by abnormalities in the synthesis of the ß chains of hemoglobin. In 2019, the United States Food and Drug Administration approved luspatercept for treating anemia in adult patients with ß-thalassemia. Other therapies, such as Janus kinase 2 (JAK2) inhibitor, have been previously reported to improve iron dysregulation and reduce synthesis of a-globin chain. Fetal hemoglobin (HbF) inducers have markedly attracted clinicians’ attention in recent years, and thalidomide was reported as a promising drug in the treatment of ßthalassemia. Thalidomide was originally used to stop vomiting in pregnancy, while it has been withdrawn from the market because of its teratogenic effects It has been reused as an immunomodulatory and anti-angiogenic drug to treat autoimmune diseases. The present meta-analysis aimed to assess the efficacy and safety of thalidomide in treating patients with ßthalassemia
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