AUTHOR=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 

TITLE=A reduced-toxicity myeloablative conditioning approach for hematopoietic cell transplant in juvenile myelomonocytic leukemia

JOURNAL=Frontiers in Oncology

VOLUME=Volume 15 - 2025

YEAR=2025

URL=https://www.frontiersin.org/journals/oncology/articles/10.3389/fonc.2025.1541192

DOI=10.3389/fonc.2025.1541192

ISSN=2234-943X

ABSTRACT=IntroductionAllogeneic 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.MethodsRetrospective study of consecutive patients with JMML who underwent HCT using a busulfan/ melphalan backbone conditioning regimen (n=17) at two academic centers.ResultsThe 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.DiscussionGiven excellent clinical outcomes at extended follow-up, prospective studies are needed to confirm our findings in this ultra-rare disease.