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单倍体相合造血干细胞移植联合人脐带间充质干细胞治疗重型再生障碍性贫血-II的临床观察

[Clinical observation of haploidentical-hematopoietic stem cell transplantation combined with human umbilical cord-derived mesenchymal stem cells for severe aplastic anemia-II].

作者信息

Xu Li-Xin, Liu Zhou-Yang, Wu Ya-Mei, Cao Yong-Bin, Li Xiao-Hong, Yan Bei, Yang Xue-Liang, DA Wan-Ming, Wu Xiao-Xiong

机构信息

Department of Hematology, The First Affiliated Hospital of Chinese PLA General Hospital, Beijing 100048, China.

Department of Hematology, Chinese PLA General Hospital, Beijing 100048, China.

出版信息

Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2014 Jun;22(3):774-8. doi: 10.7534/j.issn.1009-2137.2014.03.037.

DOI:10.7534/j.issn.1009-2137.2014.03.037
PMID:24989293
Abstract

This study was purposed to investigate the efficacy and safety of haploidentical hematopoietic stem cells (allo-HSCT) transplantation combined with human umbilical cord-derived mesenchymal stem cell infusion (hUC-MSC) for severe aplastic anemia-II (SAA-II). Eight SAA-II patients received haploidentical allo-HSCT, the G-CSF mobilized peripheral hematopoietic stem cells and bone marrow haploidentical hematopoietic stem cells were selected as graft, the human umbilical cord-derived mesenchymal stem cells (hUC-MSC) were infused as the third party. Conditioning regimen consisted of rabbit anti-thymic lymphocytes protein(ATG), cyclophosphamide(CTX) and fludarabine(Flu). For two patients out of 8 SAA-II patients the conditioning regimen was combined with busulfan(BU). The graft versus host disease(GVHD) was prevented with CSA, MTX, ATG, CD25 and mycophenolate mofetil. The results showed that the average number of nucleated cells were 9.13×10(8)/kg, and number of CD34(+)cells were 3.76×10(6)/ kg. All the 8 SAA-II patients achieved hematopoietic reconstitution. The average time of neutrophils count>0.5×10(9)/L was 11.9 days, and average time of Plt level >20×10(9)/L was 14.6 days. The incidence of acute GVHD of I-II grade was 25%, and that of III-IVgrade was 12.5%, the transplantation-related mortality was 25%. It is concluded that haploidentical allo-HSCT combined with umbilical cord MSC infusion is an effective approach to cure SAA.

摘要

本研究旨在探讨单倍型造血干细胞(allo-HSCT)移植联合人脐带间充质干细胞输注(hUC-MSC)治疗重型再生障碍性贫血II型(SAA-II)的疗效和安全性。8例SAA-II患者接受单倍型allo-HSCT,选择G-CSF动员的外周造血干细胞和骨髓单倍型造血干细胞作为移植物,输注人脐带间充质干细胞(hUC-MSC)作为第三方。预处理方案包括兔抗胸腺淋巴细胞球蛋白(ATG)、环磷酰胺(CTX)和氟达拉滨(Flu)。8例SAA-II患者中有2例预处理方案联合白消安(BU)。采用环孢素A(CSA)、甲氨蝶呤(MTX)、ATG、CD25和霉酚酸酯预防移植物抗宿主病(GVHD)。结果显示,有核细胞平均数量为9.13×10⁸/kg,CD34⁺细胞数量为3.76×10⁶/kg。8例SAA-II患者均实现造血重建。中性粒细胞计数>0.5×10⁹/L的平均时间为11.9天,血小板水平>20×10⁹/L的平均时间为14.6天。I-II级急性GVHD发生率为25%,III-IV级为12.5%,移植相关死亡率为25%。结论是单倍型allo-HSCT联合脐带间充质干细胞输注是治愈SAA的有效方法。

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