中国组织工程研究

中国组织工程研究 ›› 2015, Vol. 19 ›› Issue (50): 8095-8101.doi: 10.3969/j.issn.2095-4344.2015.50.011

• 干细胞移植 stem cell transplantation • 上一篇    下一篇

父母供者外周血单倍体移植治疗儿童难治性重型再生障碍性贫血

万鼎铭，陈晓娜，曹伟杰，邢海洲，何海燕，柳 飞，陈诗彧，庞雨晴   

  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
  • 收稿日期:2015-10-25 出版日期:2015-12-03 发布日期:2015-12-03
  • 通讯作者: 万鼎铭，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
  • 作者简介:万鼎铭，男，1963年生，河南省南阳市人，蒙古族，1998年山东医科大学毕业，博士，教授，主任医师，硕士生导师，主要从事血液病和造血干细胞移植的基础和临床研究。 并列第一作者：陈晓娜，女，1990年生，河南省新郑市人，汉族，郑州大学在读硕士，主要从事血液病和造血干细胞移植基础和临床研究。
  • 基金资助:

    211工程三期重点经费(干细胞基础与临床研究)(142-52000570)

Parental haploidentical peripheral blood stem cell transplantation for treatment of children with refractory severe aplastic anemia 

 

Wan Ding-ming, Chen Xiao-na, Cao Wei-jie, Xing Hai-zhou, He Hai-yan, Liu Fei, Chen Shi-yu,
Pang Yu-qing   

  1. Department of Hematology, the First Affiliated Hospital of Zhengzhou University, Zhengzhou 450052, Henan Province, China
  • Received:2015-10-25 Online:2015-12-03 Published:2015-12-03
  • Contact: Wan Ding-ming, Department of Hematology, the First Affiliated Hospital of Zhengzhou University, Zhengzhou 450052, Henan Province, China
  • About author:Wan Ding-ming, M.D., Professor, Chief physician, Master’s supervisor, Department of Hematology, the First Affiliated Hospital of Zhengzhou University, Zhengzhou 450052, Henan Province, China Chen Xiao-na, Studying for master’s degree, Department of Hematology, the First Affiliated Hospital of Zhengzhou University, Zhengzhou 450052, Henan Province, China
  • Supported by:

    the Funding for Phase III 211 Engineering, No. 142-52000570

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被引次数

2

摘要：

背景：在中国，儿童患者获得人类白细胞抗原相合同胞供者较困难，而以父母供者较多。
目的：回顾性分析父母供者外周血单倍体造血干细胞移植治疗儿童复发难治性重型再生障碍性贫血的疗效及安全性。
方法：纳入17例无人类白细胞抗原相合同胞及相合非血缘供者，且免疫抑制疗效不佳的复发难治性儿童重型再生障碍性贫血患者，行父母供者外周血单倍体造血干细胞移植。采用“氟达拉滨+环磷酰胺＋兔抗人胸腺细胞球蛋白抗体”预处理方案，应用环孢素+吗替麦考酚酯+甲氨蝶呤三联短程预防移植物抗宿主病。
结果与结论：①患者17例中16例(94%)获得造血重建，中性粒细胞≥0.5×109 L-1和血小板≥20×109 L-1的中位时间分别为13(11-15) d和17(12-28) d。②急性移植物抗宿主病发生率47%(8/17)，其中Ⅰ-Ⅱ度29%(5/17)，Ⅲ-Ⅳ度18%(3/17)。慢性移植物抗宿主病发生率41%(7/17)。③中位随访268(43-753) d，12例存活，总生存率为71%(12/17)，死亡5例(29%)，均为移植相关死亡，其中1例植入失败死于皮肤真菌感染，1例死于移植物抗宿主病，3例死于肺部重症感染。无患儿移植后复发。④结果显示，无人类白细胞抗原相合同胞及相合非血缘供者，且免疫抑制疗效不佳的复发难治性儿童重型再生障碍性贫血患者，父母供者外周血单倍体造血干细胞移植是一种安全有效的挽救治疗方法。 

中国组织工程研究杂志出版内容重点：干细胞；骨髓干细胞；造血干细胞；脂肪干细胞；肿瘤干细胞；胚胎干细胞；脐带脐血干细胞；干细胞诱导；干细胞分化；组织工程

关键词: 干细胞, 移植, 单倍体, 外周血, 儿童重型再生障碍性贫血, 移植物抗宿主病, 生存率, 疗效

Abstract:

BACKGROUND: For pediatric patients with aplastic anemia in China, it is difficult to find human leucocyte antigen-matched sibling donors that are mostly replaced by parental donors.
OBJECTIVE: To retrospectively analyze the clinical efficacy and safety of parental haploidentical peripheral blood hematopoietic stem cell transplantation in children with relapsed and refractory severe aplastic anemia.
METHODS: Seventeen children with relapsed and refractory severe aplastic anemia who had no matched sibling or unrelated donor and failed to respond to immunosuppressive therapy were subjected to parental haploidentical peripheral blood hematopoietic stem cell transplantation. A conditioning regimen of fludarabine+cyclophosphamide+rabbit anti-human thymocyte immunoglobulin antibody and the triple therapy of methotrexate, cyclosporine A and mycophenolate mofetil were applied to prevent graft-versus-host disease.
RESULTS AND CONCLUSION: (1) Of the 17 children, 16 cases (94%) reached hematopoietic reconstitution, and the median time of neutrophils ≥ 0.5×109/L and platelets ≥ 20×109/L was 13 (11-15) days and 17 (12-28) 
days, respectively. (2) Incidence of acute graft-versus-host disease was 47% (8 of 17 cases), including 29% (5/17) of grades I-II and 18% (3/17) of grades III-IV. Incidence of chronic graft-versus-host disease was 41% (7/17). (3) With a median follow-up duration of 268 (43-753) days, the overall survival rate was 70.6% (12/17). Five dead cases (29%) belonged to transplantation-related death, including one case of fungal skin infections, one case of graft-versus-host disease, three cases of severe lung infection. No relapse case was reported. These findings indicate that if there are no matched sibling or unrelated donors and the immunosuppression effect is poor, parental haploidentical peripheral blood hematopoietic stem cell transplantation is a safe and effective salvage treatment for children with relapsed and refractory severe aplastic anemia.
中国组织工程研究杂志出版内容重点：干细胞；骨髓干细胞；造血干细胞；脂肪干细胞；肿瘤干细胞；胚胎干细胞；脐带脐血干细胞；干细胞诱导；干细胞分化；组织工程

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