单倍体造血干细胞联合脐带间充质干细胞移植治疗重型再生障碍性贫血-Ⅰ型的临床观察

Clinical study of cotransplantation of haploidentical hematopoietic stem cells and umbilical cord mesenchymal stem cells for severe aplastic anemia-Ⅰ

  • 摘要: 目的 探讨单倍体相合造血干细胞移植联合脐带血间充质干细胞 (human umbilical cord derived mesenchymal stem cell, hUC-MSC) 治疗重型再生障碍性贫血-Ⅰ型 (severe aplastic anemia-Ⅰ, SAA-Ⅰ) 的疗效及相关并发症。 方法 14例重型再生障碍性贫血-Ⅰ型患者均为2012年1月-2013年12月在解放军总医院第一附属医院血液科确诊并进行单倍体造血干细胞移植的病例。移植物选用G-CSF动员的外周血加骨髓干细胞混合移植方案, 同时加入脐带间充质干细胞作为第三方细胞。预处理采用兔抗人T淋巴细胞球蛋白+环磷酰胺方案+福达拉滨, 移植物抗宿主病 (graft-versus-host disease, GVHD) 的预防采用环孢菌素A+霉酚酸酯+CD25单克隆单抗。 结果 14例全部获得造血重建, 白细胞恢复, 中性粒细胞>0.5×109/L平均时间10.7 d, 血小板>20×109/L平均时间13.3 d。7例发生急性移植物抗宿主病 (aGVHD), 其中Ⅰ度2例, Ⅲ度5例, 3例出现移植相关死亡。 结论 联合hUC-MSC的单倍体异基因造血干细胞移植治疗SAA-Ⅰ安全可行, 白细胞恢复快, 并发症少。

     

    Abstract: Objective To investigate the curative effect and safety of transplantation of haploidentical hematopoietic stem cells(alloHSCT) combined human umbilical cord derived mesenchymal stem cell(hUC-MSC) for severe aplastic anemia-I(SAA-I). Methods Fourteen patients who diagnosed as SAA-I in our hospital and received haploidentical allo-HSCT from June 2012 to December 2013 were enrolled in this study. Graft came from haploidentical hematopoietic bone marrow and peripheral blood mobilized with granulocyteclony stimulating factor(G-CSF), and the human umbilical cord derived mesenchymal stem cells(hUC-MSC) were infused as the third-party cells. Conditioning regimen consisted of rabbit anti-thymiclymphocytes protein(ATG), cyclophosphamide(CTX) and fludarabine(Flu), and the graft versus host disease(GVHD) was prevented with CSA, MMF, and CD25. Results All patients achieved hematopoietic reconstitution. The average time for neutrophils count>0.5×109/L was 10.7 days, and for Plt level>20×109/L was 13.3 days. Of the 7 patients had aGVHD, 2 were grade Ⅰ-Ⅱ and 5 were grade Ⅲ-Ⅳ. Three patients showed transplantation related mortality. Conclusion Haploidentical allo-HSCT combined with umbilical cord MSC infusion is a safe and effective treatment for refractory severe aplastic anemia-I with rapid hematopoietic recovery and few transplant complications.

     

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