Evidence map›Paper›PMID 40530013›Full record

ArticleFrontiers in oncology2025

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

Eman Elsabagh, Rachel Gallant, Lior Goldberg, Aditya Sharma, Paul L Martin, Timothy A Driscoll, Andrea Bauchat, Joanne Kurtzberg, LaTarsha Spencer, Paibel I Aguayo-Hiraldo and 3 more

Abstract read
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In one paragraph

Article in Frontiers in oncology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

13 authors.

Eman Elsabagh *Department of Pediatrics, Division of Transplantation and Cellular Therapy, Duke University, Durham, NC, United States.
Rachel Gallant *Division of Pediatric Hematology-Oncology, University of Oklahoma Health Sciences Center, Oklahoma City, OK, United States.
Lior Goldberg *Department of Pediatrics, T Cell Therapeutics Research Laboratories, City of Hope National Medical Center and Beckman Research Institute, Duarte, CA, United States.
Aditya Sharma *Division of Transplant/Cell Therapy and Hematological Malignancies, Cancer Center, Departments of Pediatrics and Medicine, Loma Linda University School of Medicine, Children Hospital and Medical Center, Loma Linda, CA, United States.
Paul L MartinDepartment of Pediatrics, Division of Transplantation and Cellular Therapy, Duke University, Durham, NC, United States.
Timothy A DriscollDepartment of Pediatrics, Division of Transplantation and Cellular Therapy, Duke University, Durham, NC, United States.
Andrea BauchatDepartment of Pediatrics, Division of Transplantation and Cellular Therapy, Duke University, Durham, NC, United States.
Joanne KurtzbergDepartment of Pediatrics, Division of Transplantation and Cellular Therapy, Duke University, Durham, NC, United States.
LaTarsha SpencerDepartment of Pediatrics, Division of Transplantation and Cellular Therapy, Duke University, Durham, NC, United States.
Paibel I Aguayo-HiraldoSection of Blood & Marrow Transplantation, Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, CA, United States.
Neena KapoorSection of Blood & Marrow Transplantation, Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, CA, United States.
Kris M Mahadeo *Department of Pediatrics, Division of Transplantation and Cellular Therapy, Duke University, Durham, NC, United States.
Hisham Abdel-Azim *Division of Transplant/Cell Therapy and Hematological Malignancies, Cancer Center, Departments of Pediatrics and Medicine, Loma Linda University School of Medicine, Children Hospital and Medical Center, Loma Linda, CA, United States.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

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.

Indexed as

busulfanconditioning regimenhematopoietic stem cell transplantJMMLmelphalanpediatric

Identifiers

PMID40530013
PMCPMC12170293

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