同期重型再生障碍性贫血Ⅰ型与Ⅱ型异基因外周血造血干细胞移植治疗临床疗效对比观察  

Comparative observation of clinical efficacy in treating SAAⅠand SAAⅡby allogeneic peripheral blood stem cell transplantation

作　　者：史月 冯术青 姚晶晶 姚艳红 刘志彬 张丽蕊 高峰 李晓宇 SHI Yue;FENG Shuqing;YAO Jingjing;YAO Yanhong;LIU Zhibin;ZHANG Lirui;GAO Feng;LI Xiaoyu(Department of Hematology,North China University of Science and Technology Affiliated Hospital,Tangshan 063000,China)

机构地区：[1]华北理工大学附属医院血液科,河北唐山063000

出　　处：《空军军医大学学报》2024年第5期557-562,共6页Journal of Air Force Medical University

基　　金：河北省医学科学研究计划项目(20191115,20221541)。

摘　　要：目的通过对华北理工大学附属医院同期进行的重型再生障碍性贫血(SAA)Ⅰ型(SAAⅠ)与Ⅱ型(SAAⅡ)亲缘间异基因外周血造血干细胞移植(allo-PBSCT)后的临床疗效进行对比观察,为SAA患者合理选择移植治疗时机提供帮助。方法回顾分析自2002年12月至2022年12月间在华北理工大学附属医院进行allo-PBSCT的SAAⅠ和SAAⅡ患者的临床资料,其中SAAⅠ14例,SAAⅡ28例。人类白细胞抗原同胞全相合10例(MSD组),亲缘间单倍体相合32例(HID组)。全组患者所采用的移植预处理方案均为改良白消安/环磷酰胺+兔抗人胸腺细胞免疫球蛋白方案,移植物抗宿主病预防均采用常规环孢素A(CsA)+短程甲氨蝶呤+吗替麦考酚酯,CsA血药谷浓度维持在150~300μg/L。对患者移植后总生存(OS)率及无事件生存(EFS)率进行对比观察。结果随访至2023年2月,中位随访期64(7~143)个月,全组死亡7例均为SAAⅡ患者(MSD组1例,HID组6例),全组预计3年OS率83.3%(SAAⅠ:100.0%、SAAⅡ:75.0%),两组比较差异有统计学意义(P=0.047)。HID组OS率81.3%,MSD组OS率90.0%,两组比较差异无统计学意义(P=0.541)。全组预计3年EFS率45.2%(SAAⅠ:64.3%、SAAⅡ:35.7%),两组比较差异有统计学意义(P=0.043)。HID组EFS率43.8%,MSD组EFS率50.0%,两组比较差异无统计学意义(P=0.641)。结论对有移植适应证的SAA患者,早期进行异基因造血干细胞移植治疗可改善此类患者的临床预后。Objective To compare the clinical efficacy of allogeneic peripheral blood stem cell transplantation(allo-PBSCT)between severe aplastic anemia(SAA)typeⅠ(SAAⅠ)and SAA typeⅡ(SAAⅡ)in North China University of Science and Technology Affiliated Hospital during the same period,so as to provide help for SAA patients to reasonably choose the time of transplantation treatment.Methods The clinical data of SAAⅠand SAAⅡpatients who underwent allo-PBSCT in North China University of Science and Technology Affiliated Hospital from December 2002 to December 2022 were retrospectively analyzed,including 14 cases of SAAⅠand 28 cases of SAAⅡ.There were 10 cases of human leukocyte antigen matched sibling donor(MSD group)and 32 cases of haploidentical donor(HID group).All the patients were treated with modified busulfan/cyclophosphamide+rabbit anti-human thymocyte immunoglobulin regimen,cyclosporin A(CsA)+methotrexate(short-term)+mycophenolate mofetil was used for the prevention of graft-versus-host disease,and CsA blood trough concentration was maintained at 150300μg/L.The overall survival(OS)rate and event-free survival(EFS)rate after transplantation were compared and observed.Results Follow-up to February 2023,the median follow-up time was 64(7143)months,7 patients died in the whole group,all of whom were SAAⅡpatients(1 patient in MSD group and 6 patients in HID group),the predicted 3-year OS rate of the whole group was 83.3%(SAAⅠ:100.0%,SAAⅡ:75.0%),and the difference between the two groups was statistically significant(P=0.047).The OS rate was 81.3%in HID group and 90.0%in MSD group,and there were no significant differences between the two groups(P=0.541).The predicted 3-year EFS rate of the whole group was 45.2%(SAAⅠ:64.3%,SAAⅡ:35.7%),and the difference between the two groups was statistically significant(P=0.043).The EFS rate was 43.8%in HID group and 50.0%in MSD group,and there was no significant difference between the two groups(P=0.641).Conclusion For SAA patients with transplantation indications,ear

关 键 词：重型再生障碍性贫血 慢性再生障碍性贫血 异基因外周血造血干细胞移植 

分 类 号：R556.5[医药卫生—血液循环系统疾病]