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为探讨单倍相合未去T细胞的骨髓移植治疗儿童白血病的临床疗效及可行性,对8例白血病儿童(1.9岁-9岁)接受父/母单倍体相合骨髓移植疗效进行了评价。对5例患儿采用了含全身照射(TBI)的预处理方案,它包括阿糖胞苷+环磷酰胺+全身照射(Ara-C/CTX/TBI);对另外3例不加TBI,单用马利兰+阿糖胞苷+环磷酰胺(Bu/Ara-C/CY)预处理方案。移植物抗宿主病(GVHD)的预防措施包括供者用粒细胞集落刺激因子(G-CSF)及受者接受环孢菌素A(CsA)、氨甲蝶呤(MTX)、抗胸腺细胞球蛋白(ATG)、CD25单克隆抗体和霉酚酸酯(MMF)联合治疗。结果显示:8例患者移植后均获得造血重建,中性粒细胞数大于0.5×109/L的平均天数是16天,血小板数大于20×109/L的平均天数是17天,骨髓植活的直接证据检测证实为完全供者造血。移植后急性重度GVHD的发生率低,仅1例发生急性肠道Ⅱ-Ⅲ度GVHD(1/8);5例出现慢性GVHD,其中4例为局限性慢性GVHD,1例为泛发性(合并有肺部GVHD)。在180天内无移植相关死亡,期间无致死性严重感染发生,髓系造血和免疫功能恢复较快。中位随访33个月(范围7-56月),2例死亡,均死于白血病复发,其中1例移植后22月死于供者源复发。结论:对儿童白血病进行单倍相合未去T细胞骨髓移植时采用上述预处理方案和GVHD预防措施是安全、可行、有效的。
To investigate the clinical efficacy and feasibility of haploidentical bone marrow transplantation without T cells in the treatment of childhood leukemia, 8 children with leukemia (1.9-9 years old) undergoing haploidentical bone marrow transplantation were evaluated. Pretreatment with systemic irradiation (TBI) was included in 5 patients and included cytarabine + cyclophosphamide plus whole body irradiation (Ara-C / CTX / TBI); for the other 3 infants without TBI The program was pretreated with marilan + cytarabine + cyclophosphamide (Bu / Ara-C / CY). Preventive measures for graft-versus-host disease (GVHD) include donor granulocyte colony stimulating factor (G-CSF) and recipients receiving cyclosporine A (CsA), methotrexate Protein (ATG), CD25 monoclonal antibody and mycophenolate mofetil (MMF) combination therapy. The results showed that all the 8 patients had hematopoietic reconstitution after transplantation. The average number of days when the number of neutrophils was more than 0.5 × 109 / L was 16 days. The average number of days with platelets larger than 20 × 109 / L was 17 days. Direct evidence testing confirmed complete donor hematopoiesis. The incidence of acute severe GVHD after transplantation was low, with only 1 case of acute GVHD (1/8) of the gut, and 5 cases of chronic GVHD, of which 4 were localized chronic GVHD and 1 was generalized Combined with pulmonary GVHD). No transplant-related deaths occurred within 180 days. No serious fatal infection occurred during this period. Myeloid hematopoiesis and immune function recovered rapidly. The median follow-up was 33 months (range, 7-56 months). Two patients died of leukemia relapse. One patient died of donor recurrence at 22 months after transplantation. Conclusion: It is safe, feasible and effective to adopt the above preconditioning regimen and GVHD prophylaxis when haploidentical leukemia unite to unmatured T cell bone marrow transplantation.