Evidence map›Paper›PMID 40634537›Full record

ArticleNPJ precision oncology2025

RAS pathway targeted therapy in patients with DICER1-associated sarcomas.

Lindy Zhang, Paige H R Mallinger, Serena Zhou, Stavriani C Makri, John M Gross, Calixto-Hope G Lucas, Ying S Zou, William Mize, Damon R Olson, Senna R Munnikhuysen and 5 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in NPJ precision oncology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Case Report:Frontiers in oncology · 2026
    Article
  2. Cancers · 2025
    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

15 authors.

Lindy ZhangDepartment of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Paige H R MallingerCancer and Blood Disorders, Children's Minnesota, Minneapolis, MN, USA.
Serena ZhouDepartment of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Stavriani C MakriDepartment of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
John M GrossDepartment of Pathology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Calixto-Hope G LucasDepartment of Pathology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Ying S ZouDepartment of Pathology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
William MizeDepartment of Radiology, Children's Minnesota, Minneapolis, MN, USA.
Damon R OlsonDepartment of Pathology and Laboratory Medicine, Children's Minnesota, Minneapolis, MN, USA.
Senna R MunnikhuysenDepartment of Pediatrics, University of Kentucky, Lexington, KY, USA.
Jawhar RawwasCancer and Blood Disorders, Children's Minnesota, Minneapolis, MN, USA.
Yoav MessingerCancer and Blood Disorders, Children's Minnesota, Minneapolis, MN, USA.
Kenneth S ChenDepartment of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Kris Ann P SchultzCancer and Blood Disorders, Children's Minnesota, Minneapolis, MN, USA.
Christine A PratilasDepartment of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA. cpratil1@jhmi.edu.

Funding

LAB RESEARCH TRAINING IN PEDIATRIC ONCOLOGY-HEMATOLOGYT32CA060441 · NCI · JOHNS HOPKINS UNIVERSITY · PI ALAN D FRIEDMAN · 1993 to 2026
$14.9M
NCI NIH HHS T32 CA060441The Cancer Prevention and Research Institute of Texas RP150596
6 · The paper itself

Abstract

DICER1-associated sarcomas commonly exhibit cooperating mutations involving RAS signaling pathways, but the efficacy of therapies that target these mutations is unknown. Here we report two children with DICER1 tumor predisposition who presented with DICER1-associated sarcomas with cooperating, targetable mutations in HRAS or BRAF. Both had relapsed/progressed disease despite upfront multimodal therapy and were subsequently treated with molecularly targeted agents. In the first case, mutant BRAF became amplified after dual dabrafenib/trametinib therapy, presumably as a driver of acquired resistance. In the second case, a subclonal HRAS variant at diagnosis became the predominant clone at autopsy, suggesting its importance in therapy resistance. Together, these two cases provide molecular evidence of the significance of RAS/ERK signaling in DICER1-driven tumorigenesis and highlight the potential for targeting these cooperating mutations.

Identifiers

PMID40634537
PMCPMC12241403

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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.