CAJ | 학술논문

背景：目前治疗儿童再生障碍性贫血的主要方法为强化免疫抑制治疗或干细胞移植，后者由于供者来源少而受到限制，HLA单倍体相合的异基因造血干细胞在白血病治疗中常见应用，在再生障碍性贫血治疗中较少应用。目的：探讨单倍体相合的造血干细胞移植联合胎盘来源的间充质干细胞移植治疗重型儿童再生障碍性贫血的疗效。方法：患儿，女，7岁，确诊重型再生障碍性贫血1年半，2012-07-09接受HLA单倍体相合的异基因骨髓及外周血单个核细胞联合胎盘来源间充质干细胞移植，供者为母亲。预处理采用氟达拉滨联合环磷酰胺和抗胸腺细胞球蛋白方案。结果与结论：移植后+9 d中性粒细胞>0.5×109 L-1，+12 d完成造血重建，+100 d查STR提示植入完成。移植后+8个月停用免疫抑制药物，未发生急、慢性移植物抗宿主病。患儿随访18个月，无病生存。结果表明， HLA单倍体相合的造血干细胞联合胎盘来源间充质细胞移植治疗儿童重型再生障碍性贫血是一种安全有效、值得探索的方法。
배경：목전치료인동재생장애성빈혈적주요방법위강화면역억제치료혹간세포이식，후자유우공자래원소이수도한제，HLA단배체상합적이기인조혈간세포재백혈병치료중상견응용，재재생장애성빈혈치료중교소응용。목적：탐토단배체상합적조혈간세포이식연합태반래원적간충질간세포이식치료중형인동재생장애성빈혈적료효。방법：환인，녀，7세，학진중형재생장애성빈혈1년반，2012-07-09접수HLA단배체상합적이기인골수급외주혈단개핵세포연합태반래원간충질간세포이식，공자위모친。예처리채용불체랍빈연합배린선알화항흉선세포구단백방안。결과여결론：이식후+9 d중성립세포>0.5×109 L-1，+12 d완성조혈중건，+100 d사STR제시식입완성。이식후+8개월정용면역억제약물，미발생급、만성이식물항숙주병。환인수방18개월，무병생존。결과표명， HLA단배체상합적조혈간세포연합태반래원간충질세포이식치료인동중형재생장애성빈혈시일충안전유효、치득탐색적방법。
BACKGROUND:The main therapy of severe aplastic anemia in children is immunosuppressive therapy or stem celltransplantation, but the latter one is restricted due to few donor sources. Haploidentical hematopoietic stem celltransplantation is commonly used in leukemia, but it is stil rarely reported in the treatment of aplastic anemia. OBJECTIVE:To investigate the effect of haploidentical hematopoietic stem celltransplantation combined with placenta-derived mesenchymal stem celltransplantation for children with severe aplastic anemia. METHODS:A 7-year-old girl who had been confirmed as having severe aplastic anemia for 1.5 years received a cotransplantation of haploidentical hematopoietic stem cells combined with placenta-derived mesenchymal stem cells on July 9th , 2012. The donor was her mother. The preconditioning regimen consisted of fludarabine, cyclophosphamide, and anti-thymocyte globulin. RESULTS AND CONCLUSION:Time of neutrophil recovery (>0.5×10 9/L) was+9 days, and hematopoietic reconstruction was complete at+12 days. The short tandem repeat analysis showed 100%donor’s genotype at+100 days. Immunosuppressive drugs were stopped at+8 months, and no acute or chronic graft-versus-host disease occurred. With a fol ow-up of 18 months, she was in the disease-free survival period. Our findings suggest that the cotransplantation of al ogeneic haploidentical hematopoietic stem cells and placenta-derived mesenchymal stem cells is a new effective approach for children with severe aplastic anemia, which is worth exploring in the future.