Department of Hematology, General Hospital of the Air Force, Beijing, China..
Pediatrics. 2012 Jun;129(6):e1612-5. doi: 10.1542/peds.2011-2091. Epub 2012 May 7.
We report here the preliminary results of allogeneic hematopoietic stem cell transplantation with mesenchymal stem cells (MSCs) for 6 cases of severe aplastic anemia. The patients ranged in age from 3 to 16 years, and the median time from diagnosis to transplantation was 32 months (range: 3-156 months). The conditioning regimens consisted of fludarabine, cyclophosphamide, and antithymocyte globulin with or without busulfan. Graft-versus-host disease (GvHD) was prevented by the administration of cyclosporine A, methotrexate, and mycophenolate mofetil, with or without anti-CD25 monoclonal antibody. The grafts were granulocyte colony-stimulating factor-mobilized bone marrow and peripheral blood from HLA antigen-haploidentical donors (3 cases) or peripheral blood only from unrelated HLA antigen-identical donors (3 cases). MSCs were intravenously injected at a median dose of 1.43 × 10(6)/kg (range: 0.85-2.5 × 10(6)/kg). The mean time for neutrophil and platelet recovery was 12.3 and 13.8 days, respectively. Acute GvHD grade I and II developed in 2 cases, and no chronic GvHD was documented. All patients were alive and transfusion independent at a median follow-up of 15 months (range: 6-29 months). Our report suggests that cotransplantation of allogeneic hematopoietic stem cells and MSCs might provide an opportunity for therapy for children with severe aplastic anemia.
我们在此报告 6 例重型再生障碍性贫血患者接受异基因造血干细胞移植联合间充质干细胞（MSCs）治疗的初步结果。患者年龄 3-16 岁，中位诊断至移植时间为 32 个月（范围：3-156 个月）。预处理方案包括氟达拉滨、环磷酰胺和抗胸腺细胞球蛋白，联合或不联合白消安。环孢素 A、甲氨蝶呤和吗替麦考酚酯联合或不联合抗 CD25 单克隆抗体预防移植物抗宿主病（GVHD）。移植物为 HLA 抗原单倍体相合供者（3 例）或无关 HLA 抗原相合供者（3 例）的粒细胞集落刺激因子动员骨髓和外周血，MSCs 以中位数剂量 1.43×10(6)/kg（范围：0.85-2.5×10(6)/kg）静脉输注。中性粒细胞和血小板恢复的平均时间分别为 12.3 天和 13.8 天。2 例发生 1 级和 2 级急性 GVHD，无慢性 GVHD。中位随访 15 个月（范围：6-29 个月）时，所有患者存活且无需输血。我们的报告表明，异基因造血干细胞和 MSCs 共移植可能为重型再生障碍性贫血患儿的治疗提供机会。
