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背景:儿童复发难治急性白血病单纯化学治疗效果极差,异基因造血干细胞移植是治愈该类疾病的惟一有效方法。研究显示单倍体移植与同胞相合及非亲缘全相合造血干细胞移植的治疗效果接近,甚至优于后者,且父母作为供者单倍体造血干细胞移植依从性好,能够保证移植干细胞数量及预防原发病复发,明显提高了患者移植成功率及长期无白血病生存率。目的:回顾分析父母供者单倍体外周血造血干细胞移植治疗儿童复发难治急性白血病的疗效。方法:入选35例父母供者外周血单倍体造血干细胞移植治疗儿童复发难治急性白血病。均采用“改良1,4-丁二醇二甲磺酸酯/环磷酰胺+胸腺细胞免疫球蛋白”预处理方案和环孢素、吗替麦考酚酯及甲氨蝶呤三联短程预防移植物抗宿主病。结果与结论:35例父母供者外周血单倍体造血干细胞移植治疗儿童复发难治急性白血病均植入成功。135例患儿回输单个核细胞中位数为5.82(3.23-8.45)×108/kg,其中CD34+细胞中位数为4.52(2.37-11.51)×106/kg。2干细胞回输后100 d内,移植相关死亡率为14.3%。33-Ⅱ度急性移植物抗宿主病发生率为34.3%,Ⅲ-Ⅳ度急性移植物抗宿主病发生率为37.1%,慢性移植物抗宿主病总发生率为42.9%。42年无白血病生存率为42.9%,2年总生存率为51.4%,2年原发病复发率为34.3%,中位生存时间为24个月。提示对于无人类白细胞抗原相合同胞供者及不能及时寻找到非血缘人类白细胞抗原相合供者的儿童复发难治急性白血病,父母供者外周血单倍体造血干细胞移植是一种高效可行的治疗方法。
Background: The simple chemotherapy of refractory childhood relapse acute leukemia is very poor. Allogeneic hematopoietic stem cell transplantation is the only effective way to cure such diseases. Studies have shown that haploidentical transplantation with sibling matched and unrelated full matched hematopoietic stem cell transplantation treatment effect is close to or better than the latter, and parents as donors haploidentical hematopoietic stem cell transplantation compliance, to ensure that the number of stem cell transplantation and prevention Recurrence of the primary disease, significantly improve the success rate of patients with transplantation and long-term survival of leukemia. OBJECTIVE: To retrospectively analyze the effect of haploidentical peripheral blood stem cell transplantation from parents and donors on refractory acute leukemia in children. Methods: Peripheral blood haploid hematopoietic stem cell transplantation was performed in 35 children with refractory relapsed childhood acute leukemia. Are used “modified 1,4-butanediol dimethanesulfonate / cyclophosphamide + thymocyte immunoglobulin” pretreatment program and cyclosporine, mycophenolate mofetil and methotrexate triple short range Prevention of graft versus host disease. RESULTS AND CONCLUSION: 35 cases of peripheral blood haploid hematopoietic stem cell transplantation from parents were successful in the treatment of relapsed and refractory acute leukemia in children. The median number of mononuclear cells transfused from 135 children was 5.82 (3.23-8.45) × 108 / kg, of which the median of CD34 + cells was 4.52 (2.37-11.51) × 106 / kg. 2 stem cell transplantation within 100 d after transplantation-related mortality was 14.3%. The incidence of 33-II acute graft-versus-host disease was 34.3%. The incidence of acute graft-versus-host disease (Ⅲ-Ⅳ) was 37.1%. The overall incidence of chronic graft-versus-host disease was 42.9%. 42 years without leukemia survival rate was 42.9%, 2-year overall survival rate was 51.4%, 2-year primary disease recurrence rate was 34.3%, the median survival time was 24 months. It is suggested that peripheral blood haploidentical hematopoietic stem cell transplantation from parents and donors is an effective and feasible treatment for relapse-refractory acute leukemia in non-HLA-matched sibling donors and children who can not find non-HLA-matched donor in time. .