单倍体骨髓移植治疗儿童难治复发性白血病

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目的 探讨供者应用粒细胞集落刺激因子 (G CSF)和受者联合应用多种免疫抑制剂单倍体骨髓移植治疗儿童难治复发性白血病的可行性。方法  8例难治复发性儿童白血病患儿中 ,高危急性非淋巴白血病 1例 ,急性淋巴白血病第 2次完全缓解 (CR2 )及以上的病例 6例 ,慢性粒细胞白血病加速期 1例 ,在清髓性预处理后接受单倍体供者的骨髓。供者应用G CSF 2 5 0 μg,连用 7d后采髓 ,受者移植物抗宿主病 (GVHD)的预防除环孢菌素A(CSA)和氨甲蝶呤 (MTX)外 ,在 - 4至 - 1d(移植前为“ -”)应用抗胸腺细胞球蛋白 (ATG) 5mg/ (kg·d) ,+7d(移植后为“ +”)开始加服霉酚酸酯(MMF)。结果  8例患儿均植入成功 ,中性粒细胞大于 0 5× 10 9/L和血小板大于 2 0× 10 9/L的中位天数分别是 18d(范围 16~ 2 1d)和 19 5d(范围 17~ 2 7d)。急性Ⅱ°~Ⅳ°GVHD发生 4例 (5 0 % ) ,其中 2例急性Ⅱ度肠道GVHD ,2例急性Ⅲ度肠道GVHD ,可评价的 5例患儿均发生慢性GVHD ,无一例发生广泛性慢性GVHD。中位随访时间是 5 10d(范围 390~ 72 0d) ,死亡 3例 ,其中死于急性GVHD 2例 ,死于感染 1例。无病存活 5例。结论 单倍体骨髓移植治疗儿童难治复发性白血病是一种有效和安全方法 ,对我国单亲家庭拓宽供髓来源有重要的实用价值 Objective To investigate the feasibility of donor G-CSF and recipients combined with immunosuppressive haploidentical bone marrow transplantation in refractory relapsed childhood leukemia. Methods A total of 8 children with refractory relapsed childhood leukemia were enrolled in this study. Among them, 1 were high-risk acute non-lymphatic leukemia, 6 were acute rebleeding leukemia with second complete remission (CR2) and above, 1 were chronic myeloid leukemia Myeloid preconditioning received haploid donor bone marrow. The donor was given G CSF 250 μg and used for 7 days. The prevention of graft versus host disease (GVHD) in recipients was better than that of cyclosporin A (CSA) and methotrexate (MTX) Mycophenolate mofetil (MMF) was started on day 1 (“-” before transplantation) with anti-thymocyte globulin (ATG) 5 mg / (kg · d) and +7 d (“+” after transplantation). Results All the 8 cases were successfully implanted. The median number of neutrophils larger than 0 5 × 10 9 / L and platelets larger than 2 0 × 10 9 / L were 18 days (range 16-21 days) and 195 days Range 17 ~ 2 7d). Four cases (50%) of acute Ⅱ ° ~ Ⅳ ° GVHD occurred, of which 2 cases had acute grade Ⅱ GVHD and 2 cases had acute grade Ⅲ intestinal GVHD. All five cases were evaluable, and all of them had chronic GVHD, none of which occurred Generalized chronic GVHD. The median follow-up time was 510d (range 390-72 0d), with 3 deaths, of which 2 died of acute GVHD and 1 died of infection. Disease-free survival in 5 cases. Conclusion The haploidentical bone marrow transplantation is an effective and safe method for the treatment of childhood refractory relapsed leukemia, which has important practical value for broadening the sources of marrow for single-parent families in our country
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