HLA匹配同胞供者异基因外周血干细胞移植治疗骨髓增生异常综合征138例疗效分析  

Outcomes of 138 myelodysplastic syndrome patients with HLA-matched sibling donor allogeneic hematopoietic stem cell transplantation

作　　者：王茜茜 刘子闲 赵小利 张桂新 姚剑峰 郑晓辉 张利宁 申昱妍 赵邢力 何祎 黄勇 张荣莉 魏嘉璘 马巧玲 庞爱明 杨栋林 翟卫华 姜尔烈 冯四洲 韩明哲 Wang Qianqian;Liu Zixian;Zhao Xiaoli;Zhang Guixin;Yao Jianfeng;Zheng Xiaohui;Zhang Lining;Shen Yuyan;Zhao Xingli;He Yi;Huang Yong;Zhang Rongli;Wei Jialin;Ma Qiaoling;Pang Aiming;Yang Donglin;Zhai Weihua;Jiang Erlie;Feng Sizhou;Han Mingzhe(Institute of Hematology&Blood Diseases Hospital,CAMS&PUMC,State Key Laboratory of Experimental Hematology,National Clinical Research Center for Blood Diseases,Tianjin 300020,China)

机构地区：[1]中国医学科学院血液病医院(中国医学科学院血液学研究所),实验血液学国家重点实验室,国家血液系统疾病临床医学研究中心,天津300020

出　　处：《中华血液学杂志》2020年第2期132-137,共6页Chinese Journal of Hematology

基　　金：国家自然科学基金(8167010871);天津市自然科学基金(18JCYBJC25300)。

摘　　要：目的评价人组织相容性抗原(HLA)匹配同胞供者外周血干细胞移植(MSD-PBSCT)治疗骨髓增生异常综合征(MDS)的疗效。方法回顾性总结2005年9月至2017年12月接受MSD-PBSCT治疗的138例MDS患者临床资料,观察患者总生存(OS)率、无病生存(DFS)率、复发(RR)率及非复发死亡率(NRM),分析影响移植预后的危险因素。结果①中位随访1050(4~4988)d,3年OS率、DFS率分别为(66.6±4.1)%、(63.3±4.1)%,3年累积RR率、NRM分别为(13.9±0.1)%、(22.2±0.1)%。②单因素分析显示,发生Ⅲ~Ⅳ度急性移植物抗宿主病(aGVHD)、造血干细胞移植合并症指数(HCT-CI)≥2分组和修订的国际预后积分系统(IPSS-R)极高危组的OS率均显著降低[(42.9±13.2)%对(72.9±4.2)%,χ^2=8.620,P=0.003;(53.3±7.6)%对(72.6±4.7)%,χ^2=6.681,P=0.010;(53.8±6.8)%对(76.6±6.2)%、(73.3±7.7)%,χ^2=6.337,P=0.042]。MDS伴有原始细胞过多-2(MDS-EB2)和MDS转化急性髓系白血病(MDS-AML)患者,移植前接收化疗或去甲基化治疗不改善OS[(60.4±7.8)%对(59.2±9.6)%,χ^2=0.042,P=0.838]。③多因素分析显示,HCT-CI是影响移植后OS和DFS的独立危险因素(P=0.012,HR=2.108,95%CI 1.174~3.785;P=0.008,HR=2.128,95%CI 1.219~3.712)。结论HCT-CI评价MDS患者移植后预后优于IPSS-R预后分组;发生Ⅲ~Ⅳ度aGVHD是影响移植后OS的不良预后因素;MDS-EB2和MDS-AML患者可以不化疗直接行MSD-PBSCT。Objective To evaluate the outcomes of myelodysplastic syndromes(MDS)patients who received HLA-matched sibling donor allogeneic peripheral blood stem cell transplantation(MSDPBSCT).Methods The clinical data of 138 MDS patients received MSD-PBSCT from Sep.2005 to Dec.2017 were retrospectively analyzed,and the overall survival(OS)rate,disease-free survival(DFS)rate,relapse rate(RR),non-relapse mortality(NRM)rate and the related risk factors were explored.Results①After a median follow-up of 1050(range 4 to 4988)days,the 3-year OS and DFS rates were(66.6±4.1)%and(63.3±4.1)%,respectively.The 3-year cumulative incidence of RR and NRM rates were(13.9±0.1)%and(22.2±0.1)%,respectively.②Univariate analysis showed that patients with gradeⅢ-Ⅳacute graft-versus-host disease(aGVHD)or hematopoietic cell transplantation comorbidity index(HCT-CI)≥2 points or patients in very high-risk group of the Revised International Prognostic Scoring System(IPSS-R)had significantly decreased OS[(42.9±13.2)%vs(72.9±4.2)%,χ^2=8.620,P=0.003;(53.3±7.6)%vs(72.6±4.7)%,χ^2=6.681,P=0.010;(53.8±6.8)%vs(76.6±6.2)%vs(73.3±7.7)%,χ^2=6.337,P=0.042].For MDS patients with excess blasts-2(MDS-EB2)and acute myeloid leukemia patients derived from MDS(MDS-AML),pre-transplant chemotherapy or hypomethylating agents(HMA)therapy could not improve the OS rate[(60.4±7.8)%vs(59.2±9.6)%,χ^2=0.042,P=0.838].③Multivariate analysis indicated that the HCT-CI was an independent risk factor for OS and DFS(P=0.012,HR=2.108,95%CI 1.174-3.785;P=0.008,HR=2.128,95%CI 1.219-3.712).Conclusions HCT-CI was better than the IPSS-R in predicting the outcomes after transplantation.The occurrence of gradeⅢ-ⅣaGVHD is a poor prognostic factor for OS.For patients of MDS-EB2 and MDS-AML,immediate transplantation was recommended instead of receiving pre-transplant chemotherapy or HMA therapy.

关 键 词：骨髓增生异常综合征 造血干细胞移植 同胞供者 预后因素 

分 类 号：R551.3[医药卫生—血液循环系统疾病]