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目的探讨单倍体相合造血干细胞移植联合间充质干细胞输注治疗异染性脑白质营养不良的疗效。方法 7岁患儿,采用单倍体相合造血干细胞移植联合间充质干细胞输注的方法,经氟达拉滨、马利兰、环磷酰胺和抗人胸腺细胞球蛋白方案进行预处理,供者应用G-CSF动员和联合免疫抑制剂(包括环胞菌素A、氨甲喋呤、霉酚酸酯、CD25单抗)预防移植物抗宿主病。造血重建后,每周输注脐带来源的间充质干细胞1次,连续4周。结果患儿造血重建迅速,11 d时中性粒细胞>0.5×109/L、血小板>20×109/L;14 d时血白细胞芳基硫酸酯酶A水平明显上升;28 d时达正常水平。患者无急性GVHD发生。移植后1个月经植入证据检测,证实为完全供者造血,神经系统症状逐渐恢复。结论单倍体相合造血干细胞移植,联合间充质干细胞输注,治疗异染性脑白质营养不良,安全、有效,血白细胞芳基硫酸酯酶A水平回升迅速。这种新型疗法可能是治疗缺乏HLA全相合供者的异染性脑白质营养不良疾病患者的可靠选择。
Objective To investigate the efficacy of haploidentical hematopoietic stem cell transplantation combined with mesenchymal stem cell infusion in the treatment of heterogenous leukodystrophy. Methods A 7-year-old child was pretreated with fludarabine, melilan, cyclophosphamide and anti-human thymocyte globulin by haploidentical hematopoietic stem cell transplantation combined with mesenchymal stem cell infusion. The donor application G-CSF mobilization and combined immunosuppressive agents (including cyclosporin A, methotrexate, mycophenolate mofetil and CD25 mAb) prevent graft versus host disease. After hematopoietic reconstitution, umbilical cord-derived mesenchymal stem cells were infused weekly for 4 weeks. Results The hematopoietic reconstitution in children was rapid, with neutrophils> 0.5 × 109 / L and platelets> 20 × 109 / L on day 11; the levels of aryl sulfatase A in blood leucocytes increased significantly on day 14 and reached the normal levels on day 28 . No acute GVHD occurred. Transplanted 1 month after implantation evidence testing, confirmed as complete donor hematopoietic, neurological symptoms gradually restored. Conclusions Haploid haploidentical hematopoietic stem cell transplantation combined with infusion of mesenchymal stem cells is safe and effective in the treatment of allogeneic white matter dystrophy. Arylsulfatase A levels in white blood cells rose rapidly. This new type of therapy may be a reliable option for treating patients with ALL who lack HLA total donors.