Evidence map›Paper›PMID 39808803›Full record

ReviewBlood2025

Precision medicine for high-risk gene fusions in pediatric AML: a focus on KMT2A, NUP98, and GLIS2 rearrangements.

Grace Egan, Sarah K Tasian

Abstract readReview
In one paragraph

Review in Blood, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed, 2 pooled it
–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

14 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Guideline
  2. Pooled it
  3. Refining favorable-risk classification in pediatricBlood science (Baltimore, Md.) · 2026
    Article
  4. Article
  5. Review
  6. Article
  7. Article
  8. Article
  9. Article
  10. RareAmerican journal of cancer research · 2026
    Article
  11. Article
  12. Article
  13. Article
  14. Case Report: Pediatric AML withFrontiers in oncology · 2025
    Article
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

2 authors.

Grace EganDivision of Haematology/Oncology, The Hospital for Sick Children, Toronto, ON, Canada.ORCID 0000-0002-4036-8403
Sarah K TasianDivision of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, PA.ORCID 0000-0003-1327-1662

Funding

Multispecific targeting incorporating cytokine receptor pathways in high risk pediatric acute leukemias to improve durability of adoptive cell therapy-induced remissionsU01CA232486 · NCI · UNIVERSITY OF COLORADO DENVER · PI FRY, TERRY J., TASIAN, SARAH KATHLEEN · 2018 to 2018
$4.0M
Towards rational design of combination therapeutic targetsU01CA243072 · NCI · CHILDREN'S HOSP OF PHILADELPHIA · PI TAN, KAI, TASIAN, SARAH KATHLEEN · 2020 to 2024
$2.6M
NCI NIH HHS U01 CA232486NCI NIH HHS U01 CA243072
6 · The paper itself

Abstract

abstractRobust genetic characterization of pediatric acute myeloid leukemia (AML) has demonstrated that fusion oncogenes are highly prevalent drivers of AML leukemogenesis in young children. Identification of fusion oncogenes associated with adverse outcomes has facilitated risk stratification of patients, although successful development of precision medicine approaches for most fusion-driven AML subtypes have been historically challenging. This knowledge gap has been in part due to difficulties in targeting structural alterations involving transcription factors and in identification of a therapeutic window for selective inhibition of the oncofusion without deleterious effects upon essential wild-type proteins. Herein, we discuss the current molecular landscape and functional characterization of 3 of the most lethal childhood AML fusion-oncogene driven subtypes harboring KMT2A, NUP98, or CBFA2T3::GLIS2 rearrangements. We further review early-phase clinical trial data of novel targeted inhibitors and immunotherapies that have demonstrated initial success specifically for children with these poor-prognosis genetic subtypes of AML and provide appreciable optimism to improve clinical outcomes in the future.

Indexed as

Gene RearrangementHistone-Lysine N-MethyltransferaseLeukemia, Myeloid, AcuteMyeloid-Lymphoid Leukemia ProteinNuclear Pore Complex ProteinsOncogene Proteins, FusionPrecision MedicineChildHumansHistone-Lysine N-MethyltransferaseKMT2A protein, humanMyeloid-Lymphoid Leukemia ProteinNuclear Pore Complex ProteinsNup98 protein, humanOncogene Proteins, Fusion

Identifiers

PMID39808803
PMCPMC12163908

What OpenQuestion holds

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Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.