空军军医大学学报2024,Vol.45Issue(5) :557-562.DOI:10.13276/j.issn.2097-1656.2024.05.014

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 SAAⅠ and SAAⅡ by allogeneic peripheral blood stem cell transplantation

史月 冯术青 姚晶晶 姚艳红 刘志彬 张丽蕊 高峰 李晓宇
空军军医大学学报2024,Vol.45Issue(5) :557-562.DOI:10.13276/j.issn.2097-1656.2024.05.014

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 SAAⅠ and SAAⅡ by allogeneic peripheral blood stem cell transplantation

史月 1冯术青 1姚晶晶 1姚艳红 1刘志彬 1张丽蕊 1高峰 1李晓宇1
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作者信息

  • 1. 华北理工大学附属医院血液科,河北唐山 063000
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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150~300 μg/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.

Abstract

Objective To compare the clinical efficacy of allogeneic peripheral blood stem cell transplantation(allo-PBSCT)between severe aplastic anemia(SAA)type Ⅰ(SAAⅠ)and SAA type Ⅱ(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 SAAⅠ and SAAⅡ patients 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 SAAⅠ and 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 150-300 μg/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(7-143)months,7 patients died in the whole group,all of whom were SAAⅡ patients(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,early allogeneic hematopoietic stem cell transplantation can improve their clinical prognosis.

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

Key words

severe aplastic anemia/chronic aplastic anemia/allogeneic peripheral blood stem cell transplantation

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基金项目

河北省医学科学研究项目(20191115)

河北省医学科学研究项目(20221541)

出版年

2024
空军军医大学学报
第四军医大学

空军军医大学学报

CHSSCD
影响因子:0.372
ISSN:2097-1656
参考文献量28
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