单倍型造血干细胞移植治疗幼年型粒单核细胞白血病的临床研究

摘要

Objective The study was aimed to explore the efficacy and safety of haplotype allogeneic hematopoietic stem cell transplantation (allo-HSCT)in treatment of juvenile myelomonocytic leukemia (JMML). Method From January 2011 to January 2014, six children with JMML receiving haploidentical allo-HSCT were included in the study, including 4 males and 2 females, aged from 4 to 12 years old, and the mean age was 5.6 years. The median time from diagnosis to transplantation was 6.5 months (2 to 25 months). Donors received granulocyte colony-stimulating factor mobilization. Stem cell transplantation was collected from both peripheral blood and bone marrow. CTX+FLU+ATG program was used as conditioning regimen, and combined immunosuppressive agents were used for graft-versus-host disease (GVHD) prophylaxis, including cyclosporine A, amethopterin, tacrolimus,etc. Toxic and side effects,incidence of GVHD and disease-free survival of these children after transplantation were observed. Result All patients reached hematopoietic reconstitution. The average time was 16.5 d and 18.2 d, respectively, with neutrophils ≥ 0.5×109/L and platelets ≥ 20×109/L. Implantation was confirmed by the evidence of 100% of donor hematopoiesis. With a median follow-up duration of 21.5 months (2 - 40 months), three cases developed acute GVHD, two cases showed chronic GVHD. One case died of GVHD, one died of relapse, and the remaining 4 patients remained disease-free survival. The disease-free survival rate was 66.7%. Conclusion Haploidentical hematopoietic stem cell transplantation could be chosen as a treatment method for patients with JMML in the absence of matched donors.%目的：探讨单倍型异基因造血干细胞移植（allo-HSCT）治疗幼年型粒单核细胞白血病（JMML）的疗效和安全性。方法采用亲缘单倍型相合 allo-HSCT 治疗2011年1月至2014年1月收治的6例 JMML 患儿，其中男4例，女2例，年龄4～12岁，平均5.6岁。从确诊到移植的中位时间为6.5个月（2～25个月）。供者接受粒细胞集落刺激因子动员，采用骨髓加外周血干细胞联合移植，预处理方案均采用 CTX+FLU+ATG 方案，移植物抗宿主病(GVHD）预防采用联合免疫抑制剂，包括环孢素 A、甲氨蝶呤、他克莫司等。移植后观察患儿毒副反应、GVHD 和无病生存等情况。结果全部患儿获造血重建，中性粒细胞≥0.5×109/L 及血小板≥20×109/L 的平均时间分别16.5天及18.2天，植入证据检测证实为100%为完全供者造血。中位随访时间21.5个月（2～40个月），共3例发生急性 GVHD，2例发生慢性 GVHD，GVHD 死亡1例，复发死亡1例，其余4例仍无病存活，无病生存率为67%。结论单倍体相合造血干细胞移植可能是治愈 JMML 的可行方法。