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Updated: Sep 19, 2025
Busulfan as a Myelosuppressive Agent for Generating Stable High-level Bone Marrow Chimerism in Mice
Published on: April 1, 2015
A reduced-toxicity myeloablative conditioning approach for hematopoietic cell transplant in juvenile myelomonocytic
Eman Elsabagh1, Rachel Gallant2, Lior Goldberg3
1Department of Pediatrics, Division of Transplantation and Cellular Therapy, Duke University, Durham, NC, United States.
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.
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