非血缘外周血造血干细胞移植治疗重型再生障碍性贫血的有效性  

Effectiveness of unrelated peripheral blood stem cell transplantation in the treatment of severe aplastic anemia

作　　者：张素平[1] 孙玲[1] 万鼎铭[1] 曹伟杰[1] 李丽[1] 刘长凤[1] 刘玉峰[1] 王叨[1] 郭荣[1] 姜中兴[1] 谢新生[1] Zhang Suping;Sun Ling;Wan Dingming;Cao Weijie;Li Li;Liu Changfeng;Liu Yufeng;Wang Dao;Guo Rong;Jiang Zhongxing;Xie Xinsheng(Hematopoietic Stem Cell Transplantation Center,Department of Hematology,the First Affiliated Hospital of Zhengzhou University,Zhengzhou 450052,Henan Province,China)

机构地区：[1]郑州大学第一附属医院血液科造血干细胞移植中心,河南省郑州市450052

出　　处：《中国组织工程研究》2020年第31期4994-5001,共8页Chinese Journal of Tissue Engineering Research

基　　金：河南省高等学校重点科研项目计划(18A320040),项目负责人:万鼎铭。

摘　　要：背景:重型再生障碍性贫血患者获得同胞相合造血干细胞移植供者的概率低于30%,随着近期非血缘外周血造血干细胞移植技术的不断完善,其治疗良性和恶性血液病已经达到与同胞相合造血干细胞移植相似的疗效。目的:评价非血缘外周血造血干细胞移植治疗重型再生障碍性贫血的有效性和安全性。方法:回顾性分析2014年3月至2019年9月期间接受非血缘外周血造血干细胞移植治疗的25例重型再生障碍性贫血(Ⅰ型和Ⅱ型)患者,均无同胞相合供者并且拒绝接受免疫抑制治疗。移植预处理方案为氟达拉滨+环磷酰胺+兔抗人胸腺细胞球蛋白抗体。移植物抗宿主病的预防方案为环孢素+吗替麦考酚酯+短程甲氨蝶呤。观察指标包括植入率及造血重建时间、移植物抗宿主病发生率、移植相关并发症发生率、5年预计总生存率、5年预计无病生存率。该临床研究的实施获得郑州大学第一附属医院伦理委员会批准,患者及亲属签署非血缘外周血造血干细胞移植相关知情同意书。结果与结论:①25例重型再生障碍性贫血患者中有3例移植后+28 d之内死亡,无法评价植入情况。剩余22例患者中有21例(95.4%)获得造血重建,植入成功;1例(4.6%)植入失败。中性粒细胞≥0.5×10^9 L^-1和血小板≥20×10^9 L^-1的中位时间分别为12 d(9-18 d)和13 d(10-32 d)。②植入成功的21例患者中,急性移植物抗宿主病发生率为28.6%(6/21),其中Ⅰ-Ⅱ度3例,Ⅲ-Ⅳ度3例。轻度慢性移植物抗宿主病患者仅有9.5%(2/21),无中度和重度慢性移植物抗宿主病发生。③中位随访396 d(15-1886 d),18例(72.0%)存活,5年预计总生存率为71.1%,5年预计无病生存率为65.6%。7例(28.0%)死亡,其中3例(12.0%)患者死于感染,2例(8.0%)患者死于急性肠道移植物抗宿主病,1例(4.0%)患者死于颅内出血,1例(4.0%)患者死于多器官功能衰竭。④结果显示,对于无同胞相合供者的重�BACKGROUND:The probability of obtaining sibling-matched hematopoietic stem cell transplantation donors for patients with severe aplastic anemia is less than 30%.With the recent improvement of unrelated peripheral blood stem cell transplantation,the efficacy of unrelated peripheral blood stem cell transplantation in the treatment of benign and malignant hematological diseases has reached a result similar to that of sibling-matched hematopoietic stem cell transplantation.OBJECTIVE:To evaluate the efficacy and safety of unrelated peripheral blood stem cell transplantation in the treatment of severe aplastic anemia.METHODS:A retrospective analysis was made in 25 severe aplastic anemia(type Ⅰ and type Ⅱ)patients who received unrelated peripheral blood stem cell transplantation from March 2014 to September 2019.All of them were sibling-matched hematopoietic stem cell transplantation-free patients who refused to accept immunosuppressive therapy.The transplantation pretreatment regimen was“fludarabine+cyclophosphamide+rabbit anti-human thymocyte globulin antibody”,and the prevention scheme of graft-versus-host disease was“cyclosporine+mycophenolate mofetil+short-term methotrexate”.The observation indicators included implantation rate,hematopoietic reconstitution time,incidence of graft-versus-host disease,incidence of transplantation-related complications,5-year estimated overall survival rate and 5-year estimated disease-free survival rate.This study was approved by the Ethics Committee of the First Affiliated Hospital of Zhengzhou University.Patients and their family members signed the informed consent on unrelated peripheral blood stem cell transplantation.RESULTS AND CONCLUSION:(1)Three of the 25 patients died within 28 days after hematopoietic stem cell transplantation,and the implantation could not be evaluated.Of the remaining 22 patients,21 cases(95.4%)obtained hematopoietic reconstitution and the implantation was successful;1 case(4.6%)failed.The median time of neutrophil≥0.5×10^9 L^-1 and platele

关 键 词：干细胞 再生障碍性贫血 重型 造血干细胞移植 非血缘相合 植入率 移植物抗宿主病 

分 类 号：R457[医药卫生—治疗学] R394.2[医药卫生—临床医学]