含地西他滨预处理方案的allo-HSCT治疗MDS和MDS-AML的疗效及安全性  

Efficacy and Safety of Allogeneic Hematopoietic Stem Cell Transplantation with Decitabine-containing Regimen in Myelodysplastic Syndromes and Myelodysplastic Syndromes Transformed Acute Myeloid Leukemia

作　　者：王雨欣 孙耀 谢婧 刘娜 扈江伟 乔卓青 蓝三春 赵龙 杨扬 李欲航 张斌 胡亮钉 WANG Yu-Xin;SUN Yao;XIE Jing;LIU Na;HU Jiang-Wei;QIAO Zhuo-Qing;LAN San-Chun;ZHAO Long;YANG Yang;LI Yu-Hang;ZHANG Bin;HU Liang-Ding(Senior Department of Hematology,The Fifth Medical Centre of Chinese People’s Liberation Army General Hospital;Beijing Key Laboratory of Stem Cell Therapy and Transformation Research,Beijing 100071,China)

机构地区：[1]中国人民解放军总医院第五医学中心血液病医学部,北京100071 [2]中国人民解放军总医院第五医学中心造血干细胞治疗及转化研究北京市重点实验室,北京100071

出　　处：《中国实验血液学杂志》2023年第2期522-531,共10页Journal of Experimental Hematology

基　　金：军队后勤科研重大项目(AWS17J010);国家自然科学基金(82100239,31900678)。

摘　　要：目的:评估含地西他滨(Dec)预处理方案的异基因造血干细胞移植(allo-HSCT)治疗骨髓增生异常综合征(MDS)和MDS转化的急性髓系白血病(MDS-AML)患者的疗效及安全性。方法:回顾性分析2013年4月至2021年11月本中心收治的接受allo-HSCT治疗的93例MDS和MDS-AML患者的病例特征及疗效。所有患者均予以含Dec(25 mg/m^(2)/d×3 d)的清髓预处理方案。结果:93例患者中男63例、女30例;MDS 77例,MDS-AML 16例。Ⅰ/Ⅱ级预处理相关毒性反应(RRT)发生率为39.8%,仅1例(1%)患者发生Ⅲ级RRT。91例(97.8%)患者粒系植入成功,中位植入时间为14(9-27)d;87例(93.5%)患者血小板植入成功,中位植入时间为18(9-290)d。急性移植物抗宿主病(aGVHD)、Ⅲ/Ⅳ度aGVHD发生率分别为44.2%和16.2%;慢性GVHD(cGVHD)、中重度cGVHD发生率分别为59.5%和37.1%。93例患者中54例(58%)发生移植后感染,其中肺部感染(32.3%)和血流感染(12.9%)为最常见。移植后中位随访45(0.1-108)个月,5年总生存(OS)率、无病生存(DFS)率、治疗相关死亡(TRM)率、累计复发(CIR)率分别为72.7%、68.4%、25.1%和6.5%,1年无移植物抗宿主病/无复发生存率为49.3%。预后评分相对高危组或相对低危组,伴或不伴不良预后基因突变组,伴有突变数量≥3个或<3个组患者的5年OS率均相似(>70%)。多因素分析结果显示,发生Ⅲ-Ⅳ度aGVHD为影响患者OS(P=0.008)、DFS(P=0.019)的独立危险因素。结论:含Dec预处理方案的allo-HSCT治疗MDS和MDS-AML,特别对于高危患者和伴不良预后突变的患者是可行且有效的。Objective:To evaluate the efficacy and safety of allogeneic hematopoietic stem cell transplantation(allo-HSCT)with decitabine(Dec)-conditioning regimen in the treatment of myelodysplastic syndrome(MDS)and MDS transformed acute myeloid leukemia(MDS-AML).Methods:The characteristics and efficacy data of 93 patients with MDS and MDS-AML who received allo-HSCT in our center from April 2013 to November 2021 were retrospectively analyzed.All patients were administered by myeloablative conditioning regimen containing Dec(25 mg/m^(2)/d×3 d).Results:Among the 93 patients,63 males and 30 females,were diagnosed as MDS(n=77),MDS-AML(n=16).The incidence of Ⅰ/Ⅱ grade regimen-related toxicity(RRT)was 39.8%,and Ⅲ grade RRT was only found in 1 patient(1%).Neutrophil engraftment was successful in 91(97.8%)patients after a median neutrophil engraftment time of 14(9-27)days;Successful platelet engraftment was achieved in 87(93.5%)patients,with a median engraftment time of 18(9-290)days.The incidence of acute graft versus host disease(aGVHD)and grade Ⅲ/Ⅳ aGVHD was 44.2%and 16.2%,respectively.The incidence of chronic graft versus host disease(cGVHD)and moderate-to-severe cGVHD was 59.5%and 37.1%,respectively.Of the 93 patients,54(58%)developed posttransplant infections,among which lung infection(32.3%)and bloodstream infection(12.9%)were the most common.The median follow-up after transplantation was 45(0.1-108)months.The 5-year overall survival(OS)rate,disease-free survival(DFS)rate,treatment-related mortality,and cumulative incidence of relapse were 72.7%,68.4%,25.1%,and 6.5%,respectively.And the 1-year graft-versus-host disease/relapse-free survival rate was 49.3%.The patients in different group of relative high-risk prognostic scoring or low-risk prognostic scoring,with or without poor-risk mutation(s),with mutations number≥3 or<3 had similar 5-year OS rate(more than 70%).Multivariate analysis showed that the incidence of grade Ⅲ-Ⅳ aGVHD was the independent risk factor affecting OS(P=0.008)and DFS(P=0.019).Conclusio

关 键 词：地西他滨 预处理 异基因造血干细胞移植 骨髓增生异常综合征 

分 类 号：R551.3[医药卫生—血液循环系统疾病] R733.71[医药卫生—内科学]