用于青少年骨髓单核细胞白血病造血细胞移植的低毒性清髓预处理方法。
A reduced-toxicity myeloablative conditioning approach for hematopoietic cell transplant in juvenile myelomonocytic leukemia.
作者信息
Elsabagh Eman, Gallant Rachel, Goldberg Lior, Sharma Aditya, Martin Paul L, Driscoll Timothy A, Bauchat Andrea, Kurtzberg Joanne, Spencer LaTarsha, Aguayo-Hiraldo Paibel I, Kapoor Neena, Mahadeo Kris M, Abdel-Azim Hisham
机构信息
Department of Pediatrics, Division of Transplantation and Cellular Therapy, Duke University, Durham, NC, United States.
Division of Pediatric Hematology-Oncology, University of Oklahoma Health Sciences Center, Oklahoma City, OK, United States.
出版信息
Front Oncol. 2025 Jun 3;15:1541192. doi: 10.3389/fonc.2025.1541192. eCollection 2025.
INTRODUCTION
Allogeneic hematopoietic cell transplantation (HCT) is a potentially curative treatment for most children with juvenile myelomonocytic leukemia (JMML), but overall survival remains poor at 50%. Given its rarity and heterogeneity, there is no standard HCT conditioning regimen for JMML.
METHODS
Retrospective study of consecutive patients with JMML who underwent HCT using a busulfan/ melphalan backbone conditioning regimen (n=17) at two academic centers.
RESULTS
The median age at HCT was 1.9 (range 0.7-6.0) years. At a median follow up of 7.6 (range 2.9-21.5) years, 100% disease-free (DFS) and overall survival (OS), with prompt immune reconstitution were observed. This cyclophosphamide-sparing approach was associated with no transplant related mortality.
DISCUSSION
Given excellent clinical outcomes at extended follow-up, prospective studies are needed to confirm our findings in this ultra-rare disease.
引言
异基因造血细胞移植（HCT）对大多数青少年型粒单核细胞白血病（JMML）患儿来说是一种可能治愈的治疗方法，但总体生存率仍低，仅为50%。鉴于其罕见性和异质性，目前尚无针对JMML的标准HCT预处理方案。
方法
对在两个学术中心接受以白消安/美法仑为主的预处理方案进行HCT的连续JMML患者（n = 17）进行回顾性研究。
结果
HCT时的中位年龄为1.9岁（范围0.7 - 6.0岁）。在中位随访7.6年（范围2.9 - 21.5年）时，观察到无病生存率（DFS）和总生存率（OS）均为100%，且免疫功能迅速重建。这种避免使用环磷酰胺的方法未出现与移植相关的死亡。
讨论
鉴于长期随访的良好临床结果，需要进行前瞻性研究以证实我们在这种超罕见疾病中的发现。
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