中高危骨髓增生异常综合征及其转化急性髓系白血病异基因造血干细胞移植时机与疗效的临床研究  

The clinical analysis of the efficacy and the timing of allogeneic hematopoietic stem cell transplantation(allo-HSCT)for high-risk myelodysplastic syndrome(MDS)and MDS-evolved leukemia(MDS-AML)

作　　者：宋晓露[1] 王晓刚[1] 彭也[1] 金莱[1] 陈一瑞[1] 蓝建平[1] SONG Xiao-lu;WANG Xiao-gang;PENG Ye;JIN Lai;CHEN Yi-rui;LAN Jian-ping(Department of Hematology and Hematopoietic Stem Cell Transplant Center,Zhejiang Provincial People’s Hospital,Hangzhou 310014,China)

机构地区：[1]浙江省人民医院血液病科,造血干细胞移植中心,浙江杭州310014

出　　处：《中国实用内科杂志》2021年第10期867-872,共6页Chinese Journal of Practical Internal Medicine

基　　金：浙江省自然科学基金(LY20H080009);浙江省医药卫生科技项目(2020KY015)。

摘　　要：目的探讨中高危骨髓增生异常综合征(MDS)及其转化急性髓系白血病(MDS-AML)异基因造血干细胞移植(allo-HSCT)治疗的时机和疗效。方法回顾性分析2014年2月至2020年6月于浙江省人民医院行异基因造血干细胞移植的中高危MDS患者20例及MDS-AML患者12例。预处理采用改良马利兰+环磷酰胺方案23例,氟达拉+环磷酰胺减剂量方案2例,序贯CLAG/FLAG+改良马利兰+环磷酰胺增强方案7例。结果32例患者中31例获得造血重建。中位随访14(1~59)个月,移植后预期3年总生存率(OS)(45.5±11.4)%;3年无白血病生存率(LFS)(48.5±11.4)%;3年累计复发率(8.9±6.0)%(3/31),非复发死亡率(11.8±6.4)%。急性移植物抗宿主病(aGVHD)累计发生率为(63.6%±8.6)%,其中Ⅱ~Ⅳ度aGVHD发生率为(38.0±8.7)%。慢性移植物抗宿主病(cGVHD)3年累计发生率为(35.6±11.5)%。多因素分析显示,移植前病程≥6个月是影响OS(P=0.012)及LFS(P=0.011)的独立危险因素。MDSAML患者中移植前CR组3年OS及LFS显著优于非CR组(P=0.013,P=0.018)。结论对于中高危MDS尽早alloHSCT可获得更好生存;MDS-AML患者移植前有效降低肿瘤负荷可使生存获益。Objective To evaluate the efficacy and optimize the timing of allogeneic hematopoietic stem cell transplantation(allo-HSCT)for higher-risk myelodysplastic syndromes(MDS)and MDS-evolved acute myeloid leukemia(MDS-AML).Methods A retrospective review was conducted for 20 patients with MDS and 12 ptients MDS-AML treated with alloHSCT in Zhejiang Provincial People’s Hospital between February 2014 and June 2020.Conditioning regimens included Ara-c+BUCY or BUCY in 23 cases;FLU+CTX in 2 cases;FLAG/CLAG+mBUCY+Me-CCNU in 7 cases.Results Thirtyone patients achieved sustained engraftment.After a median follow up of 14(1-59)months,the 3-year estimated overall survival(OS)was(45.5±11.4)%and the 3-year estimated leukemia-free survival(LFS)was(48.5±11.4)%.The cumulative relapse rate(RR)was(8.9±6.0)%(3/31)while non-relalse mortality(NRM)was(11.8±6.4)%.The cumulative incidence of aGVHD rate was(63.6±8.6)%,while the cumulative incidence ofⅡ-Ⅳa GVHD rate was(38.0±8.7)%.The cumulative incidence of cGVHD rate was(35.6±11.5)%.Multivariate analysis by Cox’s regression model showed the disease duration of pre-transplantation is an independent risk factor for OS and LFS.For patients with MDSAML,the differences of OS and LFS were statistically significant between CR group and non-CR group(P=0.013,P=0.018).Conclusions Early transplantation may result in a better survival for patients with higher-risk MDS.Debulking chemotherapy prior transplantation can improve survival in MDS-AML patients.

关 键 词：骨髓增生异常综合征 异基因造血干细胞移植 总生存 

分 类 号：R551.3[医药卫生—血液循环系统疾病]