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

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.

Duke Scholars

Author Timothy Alan Driscoll Pediatrics, Transplant and Cellular Therapy

Author Joanne Kurtzberg Pediatrics, Transplant and Cellular Therapy

Author Paul Langlie Martin Pediatrics, Transplant and Cellular Therapy

Author Kris Mahadeo Pediatrics, Transplant and Cellular Therapy