ORIGINAL RESEARCH article

Front. Oncol.

Sec. Pediatric Oncology

Volume 15 - 2025 | doi: 10.3389/fonc.2025.1541192

This article is part of the Research TopicCritical Complications In Pediatric Oncology and Hematopoietic Cell Transplant - Volume IIIView all 4 articles

A Reduced-Toxicity Myeloablative Conditioning approach for Hematopoietic Cell Transplant in Juvenile Myelomonocytic Leukemia

Provisionally accepted
Eman ElsabbaghEman Elsabbagh1Rachel GallantRachel Gallant2Lior GoldbergLior Goldberg3,4Aditya SharmaAditya Sharma5,6,7Paul L. MartinPaul L. Martin1Timothy DriscollTimothy Driscoll1Andrea BauchatAndrea Bauchat1Joanne KurtzbergJoanne Kurtzberg1La Tarsha SpencetrLa Tarsha Spencetr1Paibel Ixia Aguayo-HiraldoPaibel Ixia Aguayo-Hiraldo8,9Neena KapoorNeena Kapoor8,9Kris Michael MahadeoKris Michael Mahadeo1Hisham Abdel-AzimHisham Abdel-Azim5,6,7*
  • 1Department of Pediatrics, Division of Transplantation and Cellular Therapy, Duke University, Durham, California, United States
  • 2Division of Pediatric Hematology-Oncology, University of Oklahoma Health Sciences Center, Oklahoma, United States
  • 3Beckman Research Institute, City of Hope, Pasadena, California, United States
  • 4Department of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, California, United States
  • 5Loma Linda University Health Care, Loma Linda, California, United States
  • 6Loma Linda University Cancer Center, Loma Linda, California, United States
  • 7Loma Linda University Children's Hospital, Loma Linda, California, United States
  • 8Section of Blood & Marrow Transplantation, Cancer and Blood Disease Institute, Children’s Hospital Los Angeles, Los Angeles, California, United States
  • 9Keck School of Medicine, University of Southern California, Los Angeles, California, United States

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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. In this retrospective study of consecutive patients with JMML who underwent HCT using a busulfan/melphalan backbone conditioning regimen (n=17) at two academic centers, 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 cyclophosphamidesparing approach was associated with no transplant related mortality and excellent clinical outcomes at extended follow-up. Prospective studies are needed to confirm our findings in this ultra-rare disease.

Keywords: JMML, pediatric, Conditioning regimen, hematopoietic stem cell transplant, Busulfan, Melphalan

Received: 07 Dec 2024; Accepted: 12 May 2025.

Copyright: © 2025 Elsabbagh, Gallant, Goldberg, Sharma, Martin, Driscoll, Bauchat, Kurtzberg, Spencetr, Aguayo-Hiraldo, Kapoor, Mahadeo and Abdel-Azim. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

* Correspondence: Hisham Abdel-Azim, Loma Linda University Health Care, Loma Linda, 11234, California, United States

Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.

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