异基因造血干细胞移植术治疗骨髓增生异常综合征患者临床疗效的分析  

Clinical Efficacy of the MDS Patients Treated by Allo-HSCT

作　　者：王清云[1] 董玉君[1] 王倩[1] 刘微[1] 孙玉华[1] 尹玥[1] 梁赜隐[1] 许蔚林[1] 李渊[1] WANG Qing-Yun;DONG Yu-Jun;WANG Qian;LIU Wei;SUN Yu-Hua;YIN Yue;LIANG Ze-Yin;XU Wei-Lin;LI Yuan(Department of Hematology,Peking University First Hospital,Beijing 100034,China)

机构地区：[1]北京大学第一医院血液内科,100034

出　　处：《中国实验血液学杂志》2021年第1期172-180,共9页Journal of Experimental Hematology

基　　金：北京大学第一医院青年临床研究专项基金(2018CR10);北京医卫健康公益基金会科研基金(YWJKJJHKJYY-B182202);北大医学青年科技创新培育基金(BMU2020PYB007)。

摘　　要：目的:分析异基因造血干细胞移植术(allo-HSCT)治疗骨髓增生异常综合征(MDS)患者的临床疗效,并探讨移植后复发的治疗新策略。方法:选取并回顾性分析2013年4月至2019年11月于北京大学第一医院行allo-HSCT的MDS患者共72例,总结移植疗效,并对影响患者生存及复发的危险因素进行探讨。结果:72例患者中,中位随访时间37(12~111)个月,57例(79.2%)患者存活,15例(20.8%)死亡。5年总生存(OS)率、无病生存(DFS)率分别为76.6%和62.3%。IPSS-R预后危险分层、TP53突变、慢性移植物抗宿主病(cGVHD)为影响患者移植后OS的危险因素,IPSS-R预后危险分层、TP53突变、Ⅲ-Ⅳ度急性移植物抗宿主病(aGVHD)为影响患者移植后DFS的危险因素。移植后,19例(26.4%)患者发生aGVHD,5例(6.9%)患者发生Ⅲ-Ⅳ度aGVHD,25例(34.7%)患者发生cGVHD,4例(5.6%)发生广泛型cGVHD。17例(23.3%)患者在移植后出现复发,5年的累积复发率为27.5%,IPSS-R预后危险分层、TP53突变、cGVHD为影响患者复发的危险因素。复发后中位生存时间为9个月。复发后有7例患者获得无病生存,10例死亡,接受甲基化药物(HMA)联合G-CSF动员的供者淋巴细胞输注(DLI)维持治疗患者的3年OS率显著高于未接受维持治疗的患者(80.0%vs 10.0%,P=0.002)。结论:allo-HSCT是治疗中高危MDS患者的有效方法,但移植后复发仍为影响患者生存的主要因素。多次HMA联合DLI的维持治疗可能提高移植后复发患者的长期生存。Objective:To analyze clinical effectiveness of myelodysplastic syndrome(MDS) patients treated by allogeneic hematopoietic stem cell transplantation(allo-HSCT),and to investigate new therapy strategy for the treatment of relapse after allo-HSCT.Methods:72 MDS patients treated by HSCT in our hospital from April 2013 to November2019 were enrolled and analyzed retrospectively.The effect of allo-HSCT was summarized.The risk factors affecting the survival and relapse of the patients were investigated.Results:Among 72 patients,the median follow up was 37(12-111) months.57 patients survived(79.2%),while 15 patients died(20.8%).The 5-year overall survival(OS) rate and 5-year disease-free survival(DFS) rate were 76.6% and 62.3%,respectively.IPSS-R,TP53 mutation and chronic graft versus-host-disease(cGVHD) were the risk factors affecting the OS of the MDS patients after treated by alloHSCT.IPSS-R,TP53 mutation and Ⅲ-Ⅳ°acute graft versus-host-disease(aGVHD) were the risk factors affecting the DFS of the MDS patients after treated by allo-HSCT.After transplanted,19 patients(26.4%) emerged aGVHD,and 5 patients(6.9%) emerged Ⅲ-Ⅳ°aGVHD,25 patients(34.7%) emerged cGVHD,while 4 patients(5.6%)emerged extensive cGVHD.17 patients(23.3%) relapsed,and the 5-year cumulative incidence of relapse(CIR) rate was 27.5%.IPSS-R,TP53 mutation and cGVHD were the risk factors affecting the relapse of the patients.The median survival time after relapse was 9 months.There were 7 out of 17 relapsed patients survived without disease,while 10 patients died.The OS rate of patients treated with maintained hypomethylation agents(HMA) combined with G-CSF mobilized donor lymphocyte infusion(DLI) was significantly higher than the patients without HMA(80.0% vs 10.0%,P=0.002).Conclusion:Allo-HSCT is an effective therapy for intermediate and high risk MDS patients.But relapse after HSCT is still a major problem that affecting the survival of the patients.Maintenance treatment of HMA combined with DLI may improve the long-time survival of MDS patients

关 键 词：骨髓增生异常综合征 异基因造血干细胞移植 生存 复发 去甲基化药物 

分 类 号：R551.3[医药卫生—血液循环系统疾病] R392.4[医药卫生—内科学]