Evidence map›Paper›PMID 42649997›Full record

ReviewCancers2026

A New Paradigm for Pediatric AML: Improving the Pipeline for Treatments Targeting Cytogenetic and Molecular Alterations.

Camila Ayerbe, Aaron E Fan, Ryan Scanlan, Reeja Raj, Samanta Catueno, Anwesha Ray, Huber Aguirre, David McCall, Michael Roth, Miriam B Garcia and 5 more

Abstract readReview
In one paragraph

Review in Cancers, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

15 authors.

Camila AyerbeDepartment of Pediatrics, University of Texas Southwestern, Dallas, TX 77030, USA.
Aaron E FanDivision of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.ORCID 0009-0003-5394-8823
Ryan ScanlanDepartment of Pharmacy, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.ORCID 0009-0007-9762-7449
Reeja RajDivision of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.ORCID 0009-0003-8336-8766
Samanta CatuenoDivision of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.ORCID 0000-0002-0072-8603
Anwesha RayDivision of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Huber AguirreDivision of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
David McCallDivision of Pediatrics, Leukemia/Lymphoma Section, The University of Texas MD Anderson Cancer Center, Houston, TX 77070, USA.ORCID 0000-0003-2297-9670
Michael RothDivision of Pediatrics, Leukemia/Lymphoma Section, The University of Texas MD Anderson Cancer Center, Houston, TX 77070, USA.
Miriam B GarciaDivision of Pediatrics, Leukemia/Lymphoma Section, The University of Texas MD Anderson Cancer Center, Houston, TX 77070, USA.ORCID 0000-0002-2978-2267
Cesar NunezDivision of Pediatrics, Leukemia/Lymphoma Section, The University of Texas MD Anderson Cancer Center, Houston, TX 77070, USA.
Irtiza N SheikhDepartment of Pediatric Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Children's Cancer Hospital, Houston, TX 77030, USA.ORCID 0000-0003-4869-8818
Guillermo Garcia-ManeroDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Branko CuglievanDivision of Pediatrics, Leukemia/Lymphoma Section, The University of Texas MD Anderson Cancer Center, Houston, TX 77070, USA.
Amber GibsonDivision of Pediatrics, Leukemia/Lymphoma Section, The University of Texas MD Anderson Cancer Center, Houston, TX 77070, USA.ORCID 0000-0002-9146-4702

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Pediatric acute myeloid leukemia (AML) is a highly heterogeneous malignancy, with cytogenetic and molecular abnormalities playing a critical role in determining prognosis and guiding treatment decisions. Despite therapeutic advances, patients with high-risk genetic mutations and translocations continue to experience suboptimal outcomes. As new targeted therapies emerge, the treatment of pediatric AML could undergo a paradigm shift, where "one-size-fits-all" chemotherapy is no longer the only frontline approach. Identifying genetic markers inform risk stratification and have greater impact on shaping the therapeutic approach, including the integration of targeted therapies such as FLT3 and menin inhibitors into frontline therapy. Furthermore, pediatric AML treatment options are being driven by recent discoveries in adult AML, broadening their clinical trials to include pediatric patients, in part due to the RACE for Children Act that went into effect in August 2020. This review identifies the most prevalent high-risk cytogenetic lesions in pediatric AML, emphasizing their incidence, prognostic significance, and implications for clinical management. By synthesizing current research on these key genetic abnormalities and their associated therapies, we aim to provide an updated perspective on the evolving landscape of high-risk pediatric AML management that can then lead to the establishment of an agile framework to rapidly evaluate, approve, and deploy novel agents.

Indexed as

clinical trialscytogeneticshigh-riskleukemiapediatricstargeted therapies

Identifiers

PMID42649997
PMCPMC13510485

What OpenQuestion holds

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Registered trials

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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.