中国组织工程研究2016，Vol.20Issue(32)：4818-4824,7.DOI:10.3969/j.issn.2095-4344.2016.32.016
单倍型异基因造血干细胞移植后大剂量环磷酰胺诱导免疫耐受治疗儿童重型再生障碍性贫血
High-dose cyclophosphamide-induced immunogenic tolerance following haploidentical allogeneic hematopoietic stem cell transplantation in severe aplastic anemia children
摘要
Abstract
BACKGROUND:High-dose cyclophosphamide (CTX)-induced immunogenic tolerance folowing haploidentical alogeneic hematopoietic stem cel transplantation (alo-HSCT) is developed to optimize the treatment of childhood severe aplastic anemia (SAA) using haplotype alo-HSCT, providing a theoretical basis for the clinical application. <br> OBJECTIVE:To investigate the clinical efficacy and safety of the use of high-dose CTX folowing haploidentical alo-HSCT in SAA children. <br> METHODS:Clinical data from 10 children with SAA undergoing haploidentical alo-HSCT at the Department of Hematology, General Hospital of Beijing Military Area from January 2013 to January 2015 were retrospectively analyzed. Pretreatment was CTX, fludarabine, Busulfex combined with anti-human lymphocyte immune globulin used for 2 consecutive days, and then 3 days after transplantation, CTX (50 mg/kg per day) was used to induce immunogenic tolerance. Combined use of cyclosporin A, methotrexate and tacrolimus functioned as a prophylaxis for graft-versus-host disease. Another 10 SAA children who underwent synchronous HLA-identical sibling HSCT served as controls. Complications and survival in children were statisticaly analyzed in the two groups. <br> RESULTS AND CONCLUSION:In the treatment group, children were folowed up until May 2015, and the median folow-up period was 18.1 months (5-28 months). Hematopoietic reconstruction was successful in al cases, and there were three cases of graft-versus-host disease, three cases of pulmonary infection and two cases dying of pulmonary infection. In the control group, the median folow-up period was 20.7 months (6-27 months), and al the children received hematopoietic reconstruction. Additionaly, there were two cases of graft-versus-host disease, four cases of pulmonary infection, one case dying of graft-versus-host disease and one case dying of pulmonary infection in the control group. The total survival rate in each group was 80%. In summary, high-dose CTX-induced immunogenic tolerance is safe and effective for SAA children undergoing haploidentical alo-HSCT, which makes the clinical efficacy of haploidentical alo-HSCT identical to that of matched HSCT.关键词
干细胞/移植/重型/再生障碍性贫血/单倍型/异基因造血干细胞移植/环磷酰胺/免疫耐受/移植物抗宿主病/国家自然科学基金分类
医药卫生引用本文复制引用
郭智,童春,刘晓东,杨凯,何学鹏,张媛,陈鹏,楼金星,陈惠仁..单倍型异基因造血干细胞移植后大剂量环磷酰胺诱导免疫耐受治疗儿童重型再生障碍性贫血[J].中国组织工程研究,2016,20(32):4818-4824,7.基金项目
国家自然科学基金DKK-1在间充质干细胞诱导免疫耐受机制中的调控作用(31200686)；北京市首都临床特色课题诱导耐受新方法造血干细胞移植治疗重型再生障碍性贫血的临床研究 Funding:the National Natural Science Foundation of China, No.31200686 （31200686）
the Capital Clinical Project of Beijing （）
