异基因造血干细胞移植治疗儿童急性髓系白血病临床研究

Allogenetic hematopoietic stem cell transplantation for children with acute myeloid leukemia

目的评估应用异基因造血干细胞移植(allo-HSCT)治疗儿童急性髓系白血病(AML)的临床疗效及相关影响因素。方法回顾分析2002年1月至2017年11月49例确诊中、高危及复发AML行allo-HSCT患儿的临床资料,分析危险度分级、HLA分型、移植前状态、移植方式、干细胞来源及急慢性移植物抗宿主病(GVHD)等对allo-HSCT治疗效果的影响。结果49例患儿中男35例、女14例,中位年龄9岁。三年总体存活率(OS)为(59.2±7.3)%,无白血病存活率(LFS)为(50.9±7.4)%。其中第1次缓解状态移植、非血缘移植、外周血干细胞移植、中危组移植的三年LFS分别为69.8%、69.2%、73.7%、65.8%。19例死亡,分别为复发13例、严重感染5例、多器官衰竭1例。COX回归模型结果显示,急性GVHD是影响移植OS的独立危险因素(RR=3.16,95%CI:1.23~8.09,P=0.017),移植前状态为部分缓解及未缓解是影响移植LFS的独立危险因素(RR=4.76,95%CI:1.52~14.94,P=0.008;RR=5.28,95%CI:1.68~16.58,P=0.004)。结论移植前状态及急性GVHD是影响Allo-HSCT治疗儿童AML疗效的关键因素;白血病复发及感染是导致死亡的主要原因。

Objective To analyze clinical efficacy of allo-genetic stem cell transplantation (allo-HSCT) for children with acute myeloid leukemia (AML) and related factors. Methods Forty-nine children with intermediate risk group, high risk group, relapsed AML group who underwent allo-HSCT in Nanfang Hospital from January of 2002 to November of 2017 were retrospectively analyzed in risk classi fication, HLA type, status before transplantation, transplantation type, source of stem cell and acute or chronic graft versus host disease (GVHD), respectively. Results A total of 49 patients were analyzed, with a median age of 9 years, including 35 boys and 14 girls. The 3 years overall survival (OS) and leukemia free survival (LFS) was 59.2%±7.3% and 50.9%±7.4%, respectively. The 3-year LFS in first remission subgroup, non-related donor subgroup, peripheral blood stem cell transplantation subgroup and intermediate risk group is 69.8%, 69.2%, 73.7% and 65.8%, respectively. Causes of death consists of relapse (13/49,26.5%), severe infection (5/49,10.2%), and multiple organ failure(1/49, 2.0%). Cox regression analysis showed that acute GVHD (RR=3.16, 95%CI: 1.233~8.091, P=0.017) and status before transplantation (partial remission and non remission)(RR=4.76, 95%CI: 1.515~14.939, P=0.008;RR=5.28, 95%CI: 1.683~16.580, P=0.004) can signi ficantly affect the OS and LFS. Conclusion Status before transplantation and acute GVHD can affect the ef ficacy of allo-HSCT signi ficantly. The most common causes of death are relapse and infection.