单倍体造血干细胞移植治疗β-地中海贫血疗效的Meta分析

Haploidentical hematopoietic stem cell transplantation for beta-thalassemia:a meta-analysis

目的系统评价单倍体造血干细胞移植技术治疗β-地中海贫血的生存结局和安全性。方法计算机检索PubMed、EMbase、CBM、CNKI和WanFang Data数据库,搜集关于对β-地中海贫血患者进行单倍体造血干细胞移植的研究,检索时限均从2017年1月1日至2021年12月31日。由2位评价员独立筛选文献、提取资料并评价纳入研究的偏倚风险后,采用RevMan 5.4.1和Stata 16.0软件进行Meta分析。结果共纳入6个病例系列研究,包括286例患者。Meta分析结果显示:接受单倍体造血干细胞移植的β-地贫患者的总生存率和无地贫生存率分别为92.5%[95%CI(86.1%,96.1%)]和88.5%[95%CI(74.6%,95.3%)],发生Ⅲ~Ⅳ度急性GvHD和慢性GvHD的概率分别为11.5%[95%CI(6.5%,20.0%)]和23.1%[95%CI(12.3%,39.8%)],移植相关死亡率为6.5%[95%CI(3.8%,10.7%)]。结论近5年的临床研究提供了单倍体造血干细胞移植治疗β-地中海贫血的最新进展。目前NF-14-TM方案取得了较好的疗效。但总体而言,长期的疗效仍不明晰(5年及以上)。受纳入研究数量和质量的限制,尚待更多高质量的长期疗效比较研究证据。

Objective To systematically review the survival outcome and safety of haploidentical hematopoietic stem cell transplantation(haplo-HSCT)forβ-thalassemia.Methods The PubMed,EMbase,CNKI,WanFang Data and CBM databases were electronically searched to collect studies on haplo-HSCT forβ-thalassemia from January 1,2017 to December 31,2021.Two reviewers independently screened the literature,extracted data and assessed the risk of bias of the included studies.Meta-analysis was then performed by using RevMan 5.4.1 software and Stata 16.0 software.Results A total of 6 case-series studies involving 286 patients were included.The results of meta-analysis indicated that overall survival(OS)and thalassemia-free survival(TFS)forβ-thalassemia patients undergoing haplo-HSCT were 92.5%(95%CI 86.1%to 96.1%)and 88.5%(95%CI 74.6%to 95.3%),the incidence ofⅢ-Ⅳdegree acute graft versus host disease(Ⅲ-ⅣaGvHD)and chronic graft versus host disease(cGvHD)were 11.5%(95%CI 6.5%to 20.0%)and 23.1%(95%CI 12.3%to 39.8%),and the transplantation related mortality was 6.5%(95%CI 3.8%to 10.7%).Conclusion Relevant clinical studies published in the past 5 years provide the latest information and progress of haplo-HSCT forβ-thalassemia.At present,great efficacy has been shown in NF-14-TM therapeutic regimen,but the long-term efficacy remains unclear.Due to the limited quality and quantity of the included studies,more high-quality evidence from long-term comparative studies is still needed.