Objective To systematically review the survival outcome and safety of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for β-thalassemia. Methods The PubMed, EMbase, CNKI, WanFang Data and CBM databases were electronically searched to collect studies on haplo-HSCT for β-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) for β-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 of Ⅲ-Ⅳ degree acute graft versus host disease (Ⅲ-Ⅳ aGvHD) 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 for β-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.
