Long-term follow-up of haploidentical transplantation in relapsed/refractory severe aplastic anemia:a multicenter prospective study  

单倍型相合移植治疗重型再生障碍性贫血长期随访:一项多中心前瞻性研究

作　　者：Lan-Ping Xu Zheng-Li Xu Shun-Qing Wang De-Pei Wu Su-Jun Gao Jian-Min Yang Ling-Hui Xia Qi-Fa Liu Ming Jiang Hai Bai Xi Zhang Xin Wang Xiao-Jun Huang 

机构地区：[1]Peking University People's Hospital,Peking University Institute of Hematology,Beijing 100044,China [2]National Clinical Research Center for Hematologic Disease,Beijing 100044,China [3]Department of Hematology,Guangzhou First People's Hospital,Guangzhou 510180,China [4]Department of Hematology,The First Affiliated Hospital of Soochow University,Soochow 215004,China [5]Department of Hematology,The First Hospital of Jilin University,Changchun 130061,China [6]Department of Hematology,Changhai Hospital Affiliated to Second Military Medical University,Shanghai 200433,China [7]Department of Hematology,Xiehe Hospital Affiliated to Huazhong University of Science and Technology.Wuhan 430022,China [8]Department of Hematology,Nanfang Hospital Affiliated to Southern Medical University,Guangzhou 510515,China [9]Department of Hematology,The First Affiliated Hospital of Xinjiang Medical University,Urumchi 830054,China [10]Department of Hematology,General Hospital of Lanzhou Military Region of PLA,Lanzhou 730050,China [11]Medical Center of Hematology,The Second Affiliated Hospital of Army Medical University,Chongqing 400037,China [12]Department of Hematology,Shandong Provincial Hospital,Jinan 250021,China [13]Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation,Beijing 100044,China [14]Peking-Tsinghua Center for Life Sciences,Beijing 100044,China

出　　处：《Science Bulletin》2022年第9期963-970,M0004,共9页科学通报（英文版）

基　　金：supported by the Foundation for Innovative Research Groups of the National Natural Science Foundation of China(81621001);the National Natural Science Foundation of China(82100227);the Key Program of National Natural Science Foundation of China(81930004);the National Key Research and Development Program of China(2017YFA0104500)。

摘　　要：In recent decades,haploidentical stem cell transplantation(haplo-SCT)to treat severe aplastic anemia(SAA)has achieved remarkable progress.However,long-term results are still lacking.We conducted a multicenter prospective study involving SAA patients who underwent haplo-SCT as salvage therapy.Long-term outcomes were assessed,mainly focusing on survival and quality of life(QoL).Longitudinal QoL was prospectively evaluated during pretransplantation and at 3 and 5 years posttransplantation using the SF-36 scale in adults and the PedsQL 4.0 scale in children.A total of 287 SAA patients were enrolled,and the median follow-up was 4.56 years(range,3.01–9.05 years)among surviving patients.During the long-term follow-up,268 of 275 evaluable patients(97.5%)obtained sustained full donor chimerism,and 93.4%had complete hematopoietic recovery.The estimated overall survival and failure-free survival for the whole cohort at 9 years were 85.4%±2.1%and 84.0%±2.2%,respectively.Age(≥18 years)and a poorer performance status(ECOG>1)were identified as risk factors for survival outcomes.For Qo L recovery after haplo-SCT,we found that QoL progressively improved from pretransplantation to the 3-year and 5-year time points with statistical significance.The occurrence of chronic graft versus host disease was a risk factor predicting poorer QoL scores in both the child and adult cohorts.At the last followup,74.0%of children and 72.9%of adults returned to normal school or work.These inspiring long-term outcomes suggest that salvage transplantation with haploidentical donors can be routine practice for SAA patients without human leukocyte antigen(HLA)-matched donors.近年来,单倍型相合移植治疗重型再生障碍性贫血(再障)取得了很大的进展,但尚缺乏长期随访的数据.本文报道了一项前瞻性、多中心临床研究,聚焦单倍型相合移植作为二线方案治疗重型再障的长期疗效及生活质量,纵向、前瞻性评估了移植前、移植后3年和移植后5年的生活质量(成人应用SF-36量表,儿童应用PedsQL4.0量表).该项研究总共纳入287例病人,存活病人的中位随访时间4.56(3.01~9.05)年.在长期随访中,97.5%病人获得稳定的完全供者嵌合,93.4%病人获得完全造血重建.预计9年总生存率和无失败生存率85.4%±2.1%和84.0%±2.2%.移植时年龄(≥18岁)和体能状态评估(ECOG≥2分)是影响生存的预后因素.再障病人在单倍型移植后3年的生活质量较移植前有明显改善,且在移植后5年时有进一步改善.无论是儿童还是成人,中重度慢性移植物抗宿主病是影响生活质量的不良因素.末次随访时74.0%儿童和72.9%成人已恢复正常学习或工作.该研究提示在没有同胞全合供者的情况下,移植作为治疗重型再障患者的二线方案,单倍型供者可成为常规推荐.

关 键 词：Aplastic anemia HAPLOIDENTICAL Long-term follow-up Quality of life 

分 类 号：R556.5[医药卫生—血液循环系统疾病]