Investigating the Efficacy and Safety of Thalidomide for Treating Patients With ß-Thalassemia: A Meta-Analysis.
Investigating the Efficacy and Safety of Thalidomide for Treating Patients With ß-Thalassemia: A Meta-Analysis.
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DOI：
10.3389/fphar.2021.814302
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发表时间：
2021
影响因子：
5.6
通讯作者：
Liu R
中科院分区：
文献类型：
--
作者：
Lu Y;Wei Z;Yang G;Lai Y;Liu R
目前，β地中海贫血患者的主要治疗方法包括定期输血和铁螯合，但存在一些局限性。沙利度胺是一种胎儿血红蛋白 (HbF) 诱导剂，可促进 γ-珠蛋白基因表达，据报道对 β-地中海贫血有效。因此，进行这项荟萃分析是为了评估沙利度胺治疗 β 地中海贫血患者的有效性和安全性。我们检索了从建库到2021年12月1日出版的8个数据库中的相关研究。使用R 4.0.5语言编程进行荟萃分析。对检索到的文章进行筛选后，纳入 12 篇文章，共纳入 451 名患者。 Cochrane协作风险评估工具用于评估随机对照试验（RCT）的质量和偏倚风险，非随机试验则使用纽卡斯尔-渥太华量表（NOS）进行评估。沙利度胺治疗后，汇总总缓解率 (ORR) 为 85%（95% 置信区间 (CI)：80-90%），汇总完全缓解率 (CRR) 为 54%（95% 置信区间：31-76%）。与安慰剂组相比，沙利度胺组的总体缓解率（比值比= 20.4；95% CI：6.75-61.64）和完全缓解率（比值比= 20.4；95% CI：6.75-61.64）更高。治疗后血红蛋白水平和 HbF 水平有统计学显着性增加，而成人血红蛋白 (HbA) 水平、脾脏大小和血清铁蛋白无统计学显着性差异。根据ORR和CRR结果，输血依赖性地中海贫血（TDT）患者沙利度胺的疗效显着，分别为83%和52%。因此，我们分析了三项研究中的 30 名输血依赖性地中海贫血患者，发现最常见的 ß-珠蛋白基因突变是 CD41-42 (-TCTT)，而对沙利度胺的反应并未显示出与 XmnI 多态性或 CD41-42 (-TCTT) 突变有任何统计学上的显着关系。约 30% 的患者出现沙利度胺的轻微不良反应。总的来说，沙利度胺是一种相对安全有效的疗法，可减少 β 地中海贫血患者的输血需求并提高 Hb 水平。
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.
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影响因子：
2.3
作者：
Jain, Manisha;Chakrabarti, Prantar;De, Rajib
通讯作者：
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通讯作者：
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