Abstract: Objective　To investigate the long-term quality of life in children with β-thalassemia major after reciving hematopoietic stem cell transplantation (HSCT) from human leukocyte antigen (HLA)-matched sibling donor, HLA-matched unrelated donor, and HLA mismatched donor called as haploid transplantation. Methods　A total of 133 children with β-thalassemia major who underwent HSCT in Department of Stem Cell Transplantation, The First Affiliated Hospital of Guangxi Medical University from 2012 to 2019 were enrolled in the study, including 87 males and 46 females, with an average age of 5.39 years old. Both univariate analysis and multiple linear regression analysis were used to determine the factors associated with long-term quality of life after HSCT for children with β-thalassemia major. The measurement data were analyzed by independent sample t test or one-way analysis of variance, and the count data were analyzed by χ² test. Results　A total of 133 children with β-thalassemia major and their parents participated in the questionnaire survey. The incidence of acute graft-versus-host disease (GVHD) was 14.3% (19/133), and the incidence of chronic GVHD was 3.8% (5/133).The children reciving HSCT from HLA-matched unrelated donor had a higher quality of life score and lower incidences of acute and chronic GVHD and complications compared with the children reciving haploidentical stem cell transplantation (all P<0.05). Peripheral blood hematopoietic stem cell transplantation (PBSCT) or umbilical cord blood hematopoietic stem cell transplantation (UCBT), PBSCT + bone marrow transplantation (BMT), BMT, BMT+UCBT had no significant effects on quality of life (all P>0.05). Univariate analysis showed that age (B=-3.234, P<0.001), chronic GVHD (B=-19.452, P=0.001), complications (B=-23.327, P<0.001), and duration of transplantation (B=13.369, P<0.001) were associated with the quality of life in the children with β-thalassemia major after transplantation, but other factors such as gender, graft type, donor type, HLA compatibility, donor-recipient blood type compatibility, acute GVHD, and GVHD prevention regimen were not significantly associated with the quality of life (all P>0.05). Multiple linear regression analysis showed that the older the children and the more the complications, the poorer the quality of life after transplantation, and the longer the time after transplantation, the better the quality of life in children (all P<0.05). Conclusion　HSCT should be performed for children with β-thalassemia major with suitable donors as soon as possible, and attention should be paid to the control of complications and assurance of quality of life after transplantation.

Key words: HLA-matched sibling hematopoietic stem cell transplantation, HLA-matched unrelated hematopoietic stem cell transplantation, Haploid stem cell transplantation, β-thalassemia major, Long-term quality of life

摘要： 目的　了解同胞人类白细胞抗原（HLA）全合、非亲缘HLA全和及单倍体造血干细胞移植术后重型β-地中海贫血患儿长期生存质量情况。方法　选取2012—2019年于广西医科大学第一附属医院干细胞移植病区收治的不同移植类型的133例重型β-地中海贫血患儿为研究对象，其中男87例，女46例，平均年龄为5.39岁。使用单因素分析和多重线性回归分析重型β-地中海贫血患儿造血干细胞移植术后长期生存质量的相关因素。计量资料采用独立样本t检验或单因素方差分析，计数资料采用χ²检验。结果　133例重型β-地中海贫血患儿及其家长参与本次问卷调查。急性移植物抗宿主病（GVHD）发生率为14.3%（19/133），慢性GVHD发生率为3.8%（5/133）。接受非亲缘HLA全合移植的患儿生活质量评分均较高，急性GVHD、慢性GVHD和合并症发生率均低于接受单倍体造血干细胞源移植的患儿（均P<0.05）。外周血干细胞移植（PBSCT）或脐血干细胞移植（UCBT）、PBSCT+骨髓移植（BMT）、BMT、BMT+UCBT均对生活质量无明显影响（均P>0.05）。单因素分析表明年龄（B=-3.234，P<0.001）、慢性GVHD（B=-19.452，P=0.001）、并发症（B=-23.327，P<0.001）和移植时长（B=13.369，P<0.001）与移植后重型β-地中海贫血患儿生活质量相关，其他因素如性别、移植物类型、供者类型、HLA是否相合、供受者血型是否相合、GVHD预防方案、急性GVHD与患儿生活质量均无显著相关性（均P>0.05）。多元线性回归分析表示患儿年龄越大、并发症种类越多，患儿移植后生活质量越差，而随着移植后时间延长，患儿生活质量越好（均P<0.05）。结论　有合适供者重型地中海贫血患儿尽早进行异基因造血干细胞移植，注重控制移植并发症，保证患儿移植后生活质量。

关键词: 同胞全和干细胞移植, 非亲缘干细胞移植, 单倍体干细胞移植, 重型β-地中海贫血, 长期生活质量