Evidence map›Paper›PMID 23526739›Full record

Trial reportPediatric blood & cancer2013

PAX-FOXO1 fusion status drives unfavorable outcome for children with rhabdomyosarcoma: a children's oncology group report.

Stephen X Skapek, James Anderson, Frederic G Barr, Julia A Bridge, Julie M Gastier-Foster, David M Parham, Erin R Rudzinski, Timothy Triche, Douglas S Hawkins

Abstract readMulticenter StudyRandomized Controlled Trial
In one paragraph

Trial report in Pediatric blood & cancer, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 135 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
135citing papers in PubMed, 1 pooled it
15.2field-weighted citation impact, top 1% of its field
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

135 citing papers in PubMed, 1 synthesis or guideline pooled it, 271 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Trial
  4. Trial
  5. Trial
  6. Trial
  7. Observational
  8. Article
  9. Pediatric Rhabdomyosarcoma in the Head and Neck: An Overview.Journal of neurological surgery reports · 2026
    Article
  10. Article
  11. Gene Amplification in Rhabdomyosarcoma: Lessons from a Rare Cancer.International journal of molecular sciences · 2026
    Review
  12. Article
  13. Article
  14. Review
  15. Article
  16. Review
  17. Article
  18. Article
  19. Article
  20. Review

75 more citing papers are in PubMed but not listed here.

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

9 authors at 8 institutions in 1 country.

Stephen X SkapekChildren's Medical Center and University of Texas Southwestern Medical Center, Dallas, Texas 75390, USA. stephen.skapek@utsouthwestern.edu
James Anderson
Frederic G Barr
Julia A Bridge
Julie M Gastier-Foster
David M Parham
Erin R Rudzinski
Timothy Triche
Douglas S Hawkins
University of Nebraska Medical Center · USChildren's Hospital of Los Angeles · USNational Cancer Institute · USNationwide Children's Hospital · USSeattle Children's Hospital · USThe University of Texas Southwestern Medical Center · USUniversity of Oklahoma Health Sciences Center · USUniversity of Washington · US

Funding

COG FOREIGN ACCRUALU10CA098543 · NCI · NATIONAL CHILDHOOD CANCER FOUNDATION · PI ADAMSON, PETER C. · 2003 to 2013
$335.5M
Children's Oncology Group Statistics &Data Center GrantU10CA098413 · NCI · UNIVERSITY OF NEBRASKA MEDICAL CENTER · PI DEVIDAS, MEENAKSHI · 2003 to 2013
$67.5M
Studies of gene fusions in rhabdomyosarcomaZIABC011387 · NCI · DIVISION OF BASIC SCIENCES - NCI · PI BARR, FREDERIC · 2011 to 2025
$9.1M
Translation of Predictive Cancer Biomarkers into Clinical PracticeRC2CA148216 · NCI · CHILDREN'S HOSPITAL OF LOS ANGELES · PI SKAPEK, STEPHEN X, TRICHE, TIMOTHY J. · 2009 to 2010
$2.9M
COG studies of gene amplification in rhabdomyosarcomaR01CA104896 · NCI · UNIVERSITY OF PENNSYLVANIA · PI BARR, FREDERIC G · 2005 to 2009
$1.8M
COG NCTN Solid Malignancy Integrated Translational Science CenterU10CA180884 · NCI · CHILDREN'S HOSP OF PHILADELPHIA · PI ADAMSON, PETER C., SKAPEK, STEPHEN X · 2014 to 2018
$1.8M
Intramural NIH HHSNCI NIH HHS CA098413NCI NIH HHS CA104896NCI NIH HHS CA148216NCI NIH HHS RC2 CA148216NCI NIH HHS U10 CA098413NCI NIH HHS U10 CA098543NCI NIH HHS U10 CA180884
6 · The paper itself

Abstract

backgroundRhabdomyosarcoma (RMS) is divided into two major histological subtypes: alveolar (ARMS) and embryonal (ERMS), with most ARMS expressing one of two oncogenic genes fusing PAX3 or PAX7 with FOXO1 (P3F and P7F, respectively). The Children's Oncology Group (COG) carried out a multi-institutional clinical trial to evaluate the prognostic value of PAX-FOXO1 fusion status.

methodsStudy participants were treated on COG protocol D9803 for intermediate risk ARMS or ERMS using multi-agent chemotherapy, radiotherapy, and surgery. Central diagnostic pathology review and molecular testing for fusion genes were carried out on prospectively collected specimens. Event-free (EFS) and overall survival (OS) at 5 years were correlated with histological subtype and PAX-FOXO1 status.

resultsOf 616 eligible D9803 enrollees, 434 cases had adequate clinical, molecular, and pathology data for definitive classification as ERMS, ARMS P3F+ or P7F+, or ARMSn (without detectable fusion). EFS was worse for those with ARMS P3F+ (54%) and P7F+ (65%) than those with ERMS (77%; P < 0.001). EFS for ARMSn and ERMS were not statistically different (90% vs. 77%, P = 0.15). ARMS P3F+ had poorer OS (64%) than ARMS P7F+ (87%), ARMSn (89%), and ERMS (82%; P = 0.006).

conclusionsARMSn has an outcome similar to ERMS and superior EFS compared to ARMS with either P3F or P7F, when given therapy designed for children with intermediate risk RMS. This prospective analysis supports incorporation of PAX-FOXO1 fusion status into risk stratification and treatment allocation.

Indexed as

RhabdomyosarcomaAdolescentAdultAntineoplastic Combined Chemotherapy ProtocolsChildChild, PreschoolDisease-Free SurvivalFemaleHumansInfantMaleNeoplasm StagingOncogene Proteins, FusionPaired Box Transcription FactorsPAX7 Transcription FactorProspective StudiesOncogene Proteins, FusionPaired Box Transcription FactorsPAX3-FOXO1A fusion protein, humanPAX7 protein, humanPAX7 Transcription FactorPAX-FOXO1rhabdomyosarcomasurvival

Identifiers

PMID23526739
PMCPMC4646073
OpenAlexW2101405991

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.