Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for juvenile myelomonocytic leukemia (JMML), but few large studies of HSCT for JMML exist. Using data from the Japan Society for Hematopoietic Cell Transplantation registry, we analyzed the outcomes of 129 children with JMML who underwent HSCT between 2000 and 2011. The 5-year overall survival (OS) rate and cumulative incidence of relapse were 64% and 34%, respectively. A regimen of busulfan/fludarabine/melphalan was the most commonly used (59 patients) and provided the best outcomes; the 5-year OS rate reached 73%, and the cumulative incidences of relapse and transplantation-related mortality were 26% and 9%, respectively. In contrast, the use of the irradiation-based myeloablative regimen was the most significant risk factor for OS (hazard ratio [HR], 2.92; P = .004) in the multivariate model. In addition, chronic graft-versus-host disease (GVHD) was strongly associated with lower relapse (HR, 0.37; P = .029) and favorable survival (HR, 0.22; P = .006). The current study has shown that a significant proportion of children with JMML can be cured with HSCT, especially those receiving the busulfan/fludarabine/melphalan regimen. Based on the lower relapse and better survival observed in patients with chronic GVHD, additional treatment strategies that focus on enhancing graft-versus-leukemia effects may further improve survival.

中文翻译：

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少年骨髓单核细胞白血病儿童同种异体造血干细胞移植后的临床结果：日本造血细胞移植学会的一份报告。

造血干细胞移植（HSCT）是治疗少年骨髓单核细胞白血病（JMML）的唯一方法，但目前尚无针对HSMM进行JMML的大型研究。我们使用了日本造血细胞移植学会登记的数据，对129名2000年至2011年接受HSCT的JMML儿童的结局进行了分析。5年总生存率（OS）和复发的累积发生率分别为64％和34％ ， 分别。最常使用白消安/氟达拉滨/美法仑的治疗方案（59例患者），并提供最佳结果。5年OS率达到73％，复发和移植相关死亡率的累积发生率分别为26％和9％。相反，使用基于放射的清髓疗法是OS的最重要危险因素（危险比[HR]为2.92； P =。004）。此外，慢性移植物抗宿主病（GVHD）与较低的复发率（HR，0.37； P = .029）和良好的生存率（HR，0.22； P = .006）密切相关。目前的研究表明，HSCT可以治愈很大一部分JMML儿童，尤其是接受白消安/氟达拉滨/美法仑治疗的儿童。基于在慢性GVHD患者中观察到的更低的复发率和更好的生存率，着重于增强移植物抗白血病作用的其他治疗策略可能会进一步改善生存率。目前的研究表明，HSCT可以治愈很大一部分JMML儿童，尤其是接受白消安/氟达拉滨/美法仑治疗的儿童。基于在慢性GVHD患者中观察到的更低的复发率和更好的生存率，着重于增强移植物抗白血病作用的其他治疗策略可能会进一步改善生存率。目前的研究表明，HSCT可以治愈很大一部分JMML儿童，尤其是接受白消安/氟达拉滨/美法仑治疗的儿童。基于在慢性GVHD患者中观察到的更低的复发率和更好的生存率，着重于增强移植物抗白血病作用的其他治疗策略可能会进一步改善生存率。