异基因造血干细胞移植治疗骨髓增生异常综合征伴原始细胞增多282例生存分析及WHO 2022分类不同亚型的生存比较  

Analysis of the efficacy and prognostic factors of allogeneic hematopoietic stem cell transplantation in patients with myelodysplastic syndrome with blastomycosis and survival comparison of different subtypes after the WHO 2022 reclassification

作　　者：王慧 马润芝[1,2] 庞爱明 杨栋林 陈欣 张荣莉 魏嘉璘 马巧玲 翟卫华 何祎 姜尔烈 韩明哲 冯四洲 Wang Hui;Ma Runzhi;Pang Aiming;Yang Donglin;Chen Xin;Zhang Rongli;Wei Jialin;Ma Qiaoling;Zhai Weihua;He Yi;Jiang Erlie;Han Mingzhe;Feng Sizhou(State Key Laboratory of Experimental Hematology,National Clinical Research Center for Blood Diseases,Haihe Laboratory of Cell Ecosystem,Institute of Hematology&Blood Diseases Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Tianjin 300020,China;Tianjin Institutes of Health Science,Tianjin 301600,China;The Affiliated Yantai Yuhuangding Hospital of Qingdao University,Yantai 264000,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),北京协和医学院,血液与健康全国重点实验室,国家血液系统疾病临床医学研究中心,细胞生态海河实验室,天津300020 [2]天津医学健康研究院,天津301600 [3]青岛大学附属烟台毓璜顶医院,烟台264000

出　　处：《中华血液学杂志》2024年第5期445-452,共8页Chinese Journal of Hematology

基　　金：中国医学科学院医学与健康科技创新工程(2021-I2M-C&T-B-080、2021-I2M-1-017);天津市科技计划项目(21JCZDJC01170);细胞生态海河实验室创新基金(22HHXBSS00036)。

摘　　要：目的评估异基因造血干细胞移植(allo-HSCT)治疗骨髓增生异常综合征伴原始细胞增多(MDS-EB)的疗效和预后影响因素,比较WHO2022分类不同亚型患者的预后。方法纳入2006年10月至2022年12月在中国医学科学院血液病医院接受allo-HSCT的282例MDS-EB患者,按照WHO 2022诊断标准重新分类为骨髓增生异常肿瘤伴原始细胞增多1型/2型(MDS-IB1/IB2)(222例)、MDS伴纤维化(MDS-f)(41例)和伴双等位基因TP53突变的MDS(MDS-biTP53)(19例)三组,对其临床资料进行回顾性分析。结果①282例患者中位年龄46(15~66)岁,男191例,女91例,MDS-EB1118例(42%),MDS-EB2164例(58%)。②282例MDS-EB患者中256例(90.8%)移植后获得造血重建,原发植入功能不良11例(3.9%),继发植入功能不良15例(5.3%)。移植后100 d急性移植物抗宿主病(GVHD)累积发生率为(42.6±3.0)%,Ⅱ~Ⅳ度急性GVHD累积发生率为(33.0±2.8)%;移植后1年慢性GVHD累积发生率为(31.0±2.9)%。移植后128例(45.4%)患者发生巨细胞病毒(CMV)感染,63例(22.3%)患者发生菌血症,35例(12.4%)患者发生肺部真菌感染,17例(6.0%)患者发生EB病毒感染。③移植后中位随访时间为22.1(19.2~24.7)个月,3年总生存(OS)率、无病生存(DFS)率分别为71.9%(95%CI 65.7%~78.6%)、63.6%(95%CI 57.2%~70.7%),3年非复发死亡率(NRM)为17.9%(95%CI 13.9%~22.9%),3年累积复发率(CIR)为9.8%(95%CI 6.7%~13.7%)。影响移植后OS的独立危险因素包括单体核型(MK)(P=0.004,HR=3.26,95%CI 1.46~7.29)、造血干细胞移植合并症指数(HCI-CI)≥3分(P<0.001,HR=2.86,95%CI 1.72~4.75)、发生Ⅱ~Ⅳ度肠道急性GVHD(P<0.001,HR=5.94,95%CI 3.50~10.10)。④MDS-IB1/IB2组移植后3年OS率、DFS率均优于MDS-biTP53组[OS:72.0%(95%CI 63.4%~80.7%)对46.4%(95%CI 26.9%~80.1%),P=0.020;DFS:67.4%(95%CI 60.3%~75.3%)对39.7%(95%CI 22.3%~70.8%),P=0.015],3年CIR低于MDS-biTP53组[7.3%(95%CI 4.3%~11.4%)对26.9%(95%CI 9.2%~48.5%),P=0.004]。MDS-IB1/IB2组、MDS-f组、MDS-biTP53组移植后3年NRM分别为16.7%(95%CI Objective To evaluate the efficacy and prognostic factors of allogeneic hematopoietic stem cell transplantation(allo-HSCT)in patients with myelodysplastic syndrome accompanied by myelodysplasia(MDS-EB)and to compare the prognosis of different subtypes of patients classified by World Health Organization(WHO)2022.Methods A total of 282 patients with MDS-EB who underwent allo-HSCT at the Hematology Hospital of the Chinese Academy of Medical Sciences from October 2006 to December 2022 were included in the study.The WHO 2022 diagnostic criteria reclassified MDS into three groups:myelodysplastic tumors with type 1/2 of primitive cell proliferation(MDS-IB1/IB2,222 cases),MDS with fibrosis(MDS-f,41 cases),and MDS with biallelic TP53 mutation(MDS-biTP53,19 cases).Their clinical data were retrospectively analyzed.Results①The median age of 282 patients was 46(15-66)years,with 191 males and 91 females.Among them,118(42%)and 164(58%)had MDS-EB1 and MDS-EB2,respectively.②Among the 282 patients,256(90.8%)achieved hematopoietic reconstruction after transplantation,with 11(3.9%)and 15(5.3%)having primary and secondary implantation dysfunctions,respectively.The cumulative incidence of acute graft-versus-host disease(GVHD)100 days post-transplantation was(42.6±3.0)%,and the cumulative incidence of gradeⅡ-Ⅳacute GVHD was(33.0±2.8)%.The cumulative incidence of chronic GVHD 1 year post-transplantation was(31.0±2.9)%.Post-transplantation,128(45.4%),63(22.3%),35(12.4%),and 17 patients(6.0%)developed cytomegalovirus infection,bacteremia,pulmonary fungal infection,and Epstein-Barr virus infection.③The median follow-up time post-transplantation was 22.1(19.2-24.7)months,and the 3-year overall survival(OS)and disease-free survival(DFS)rates were 71.9%(95%CI 65.7%-78.6%)and 63.6%(95%CI 57.2%-70.7%),respectively.The 3-year non-recurrent mortality rate(NRM)is 17.9%(95%CI 13.9%-22.9%),and the 3-year cumulative recurrence rate(CIR)is 9.8%(95%CI 6.7%-13.7%).The independent risk factors affecting OS post-transplantation include monocy

关 键 词：骨髓增生异常综合征 骨髓增生异常肿瘤 异基因造血干细胞移植 预后 

分 类 号：R733[医药卫生—肿瘤]