Objective: To investigate the safety and efficacy of haploidentical hematopoietic stem cell transplantation with different intensity conditioning regimen in the treatment of childhood aplastic anemia (AA) . Methods: Thirty-seven AA patients who underwent haploidentical transplantation in BaYi Children's Hospital Affiliated to PLA Army General Hospital from January 2013 to January 2017 were enrolled. According to the dosage of conditioning regimen, 34 patients excluding 3 other conditioning regimens were divided into high-dosage group (regimen 2, 22 cases) and low-dosage group (regimen 3, 12 cases). The data of Engraftment, graft-vs-host disease (GVHD), hematopoietic reconstitution, relapse, infection, overall survival (OS) were analyzed. The comparison between the two groups was tested by χ(2) test. Results: A total of 35 of 37 patients achieved primary engraftment; 2 cases died of regimen-related toxicity and severe infection before the infusing of the grafts. The activation rate of CMV and EBV was 60% (21/35) . Post-transplant lymphocyte disease (PTLD) of lung occurred in one case. The cumulative incidences of acute GVHD grade Ⅰ-Ⅳ and chronic GVHD were 29% (10/35) and 34% (12/35) respectively and the incidence of extensive chronic GVHD was 6% (2/35) . The median follow-up time was 18.8 (2.9-44.1) months, the OS was 92% (34/37) .All survived patients were no longer dependent on blood transfusion and none of them had recurrence. Comparing the rates of overall survival(86%(19/22) vs.100%(12/12)) and rates of chronic GVHD(40%(8/20) vs. 17%(2/12)) in regimen 2 and regimen 3 group, there were no significant difference (χ(2)=1.742, 1.841, all P>0.05) . Significant difference was found at the incidence of Ⅰ-Ⅳ acute GVHD (10% (2/20) vs. 50% (6/12) ,χ(2)=6.200, P=0.013). Conclusions: Haploidentical hematopoietic stem cell transplantation is effective and safe. It is suitable for patients who are not eligible for matched donor transplantation. Application of reduced dose preconditioning in haploid transplantation is worth exploring.
目的： 评估不同预处理方案用于单倍体造血干细胞移植治疗儿童再生障碍性贫血（再障）的安全性和有效性。 方法： 回顾性分析2013年1月至2017年1月在陆军总医院附属八一儿童医院接受单倍体造血干细胞移植37例再障患儿的临床资料，除3例为其他预处理方案外，余34例患儿根据预处理方案中环磷磷胺及白消安的剂量，分成高剂量组22例，低剂量组12例，对植入、移植物抗宿主病（GVHD）、造血重建、复发、感染、总生存等情况进行分析，两组之间的比较采用χ(2)检验。 结果： 37例患儿中35例完成单倍体移植，全部获得完全植入，2例患儿在移植前死于预处理相关毒性和感染。巨细胞病毒/EB病毒（CMV/EBV）激活率为60%（21/35），合并1例迟发型肺部淋巴细胞增殖性疾病（PTLD），Ⅰ~Ⅳ度急性GVHD累积发生率为29%（10/35），慢性GVHD累积发生率为34%（12/35），其中广泛型慢性GVHD发生率为6%（2/35）。随访18.8（2.9~44.1）个月，总生存率为92%（34/37），生存患儿全部脱离输血，无复发病例。比较高剂量组和低剂量组的总生存率[86%（19/22）比100%（12/12）]，慢性GVHD累积发生率[40%（8/20）比17%（2/12）]，差异均无统计学意义（χ(2)=1.742、1.841，P均>0.05）,Ⅰ~Ⅳ度急性GVHD的发生率分别为10%（2/20）和50%（6/12），差异有统计学意义（χ(2)=6.200，P=0.013）。 结论： 单倍体造血干细胞移植在儿童再障的治疗中疗效肯定，安全性良好，作为缺乏全相合供者且急需移植的再障患儿的合适选择，减低剂量预处理用于单倍体移植值得深入探索。.
Keywords: Anemia, aplastic; Haploidy; Hematopoietic stem cell transplantation; Transplantation conditioning.