Evidence map›Paper›PMID 38627882›Full record

Trial reportPediatric blood & cancer2024

Event-free survival in relapsed and refractory rhabdomyosarcoma treated on cooperative group phase II trials: A report from the Children's Oncology Group.

Jonathan Metts, Wei Xue, Zhengya Gao, Sapna Oberoi, Aaron R Weiss, Rajkumar Venkatramani, Douglas J Harrison

Open access · bronzeAbstract readClinical Trial, Phase II
In one paragraph

Trial report in Pediatric blood & cancer, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed, 1 pooled it
2.9field-weighted citation impact, top 9% 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

6 citing papers in PubMed, 1 synthesis or guideline pooled it, 7 citations in OpenAlex.

  1. Guideline
  2. Trial
  3. Trial
  4. Review
  5. Review
  6. 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

7 authors at 6 institutions in 2 countries.

Jonathan MettsSarcoma Department, Moffitt Cancer Center, Tampa, Florida, USA.ORCID 0000-0003-1068-7857
Wei XueDepartment of Biostatistics, University of Florida, Gainesville, Florida, USA.
Zhengya GaoDepartment of Biostatistics, University of Florida, Gainesville, Florida, USA.
Sapna OberoiDepartment of Pediatric Hematology/Oncology, CancerCare Manitoba, Winnipeg, Manitoba, Canada.ORCID 0000-0002-6071-0042
Aaron R WeissDepartment of Pediatrics, Maine Medical Center, Portland, Maine, USA.ORCID 0000-0001-8352-3963
Rajkumar VenkatramaniDepartment of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
Douglas J HarrisonDepartment of Pediatrics, University of Texas MD Anderson Cancer Center, Houston, Texas, USA.ORCID 0000-0002-6776-4852
University of Florida · USBaylor College of Medicine · USCancerCare Manitoba · CAJohns Hopkins University · USMaine Medical Center · USThe University of Texas MD Anderson Cancer Center · US

Funding

NCTN BIQSFP ANBL1531 (NRT)U10CA180886 · NCI · PUBLIC HEALTH INSTITUTE · PI Douglas S. Hawkins · 2014 to 2026
$390.6M
COG SDMC - Statistics CoreU10CA180899 · NCI · UNIVERSITY OF SOUTHERN CALIFORNIA · PI TODD A ALONZO · 2014 to 2026
$132.8M
PEP-CTN EHR to RAVE supplementUM1CA228823 · NCI · PUBLIC HEALTH INSTITUTE · PI Elizabeth Fox · 2018 to 2026
$39.6M
Pediatric Phase 1/Pilot ConsortiumUM1CA097452 · NCI · CHILDREN'S HOSP OF PHILADELPHIA · PI WEIGEL, BRENDA J. · 2009 to 2018
$17.7M
MaineHealth Cancer Care Network (MHCCN) and NCORP Activities across the LifespanUG1CA239771 · NCI · MAINEHEALTH · PI Leslie Bradford, Christine Lu-Emerson · 2019 to 2026
$7.9M
Cookies for Kid's CancerFriends of TJ FoundationMaddie's Promise FoundationNCI NIH HHS U10 CA180886NCI NIH HHS U10 CA180899NCI NIH HHS UG1 CA239771NCI NIH HHS UM1 CA097452NCI NIH HHS UM1 CA228823NIH HHSSebastian Strong FoundationSt Baldrick's Foundation U10CA180886St Baldrick's Foundation U10CA180899St Baldrick's Foundation UM1CA097452St Baldrick's Foundation UM1CA228823Summer's Way FoundationTakeda Pharmaceuticals
6 · The paper itself

Abstract

backgroundNovel therapies are needed for relapsed and refractory rhabdomyosarcoma (RRMS). Phase II clinical trials in RRMS have typically utilized radiologic response as the primary activity endpoint, an approach that poses several limitations in RRMS. In this analysis, we aimed to estimate an event-free survival (EFS) endpoint for RRMS that could be used as a benchmark for future studies. PROCEDURE: We performed a retrospective study of patients with RRMS enrolling on 13 single-agent phase II Children's Oncology Group and legacy group trials from 1997 to 2016. All included trials used radiographic response as their primary activity endpoint. Six-month EFS was estimated from time of trial enrollment with 95% confidence intervals. Clinical characteristics, including trial of enrollment, sex, age, race, histology, number of prior chemotherapies, and radiographic response were evaluated for their impact on 6-month EFS.

resultsWe identified 175 patients across 13 trials. The 6-month EFS was 16.8% (11.6%-22.8%). No differences were seen in 6-month EFS based on age, sex, race, or histology. There were nonsignificant trends toward improved 6-month EFS for patients with less than or equal to two prior lines of therapy versus higher than two, for patients enrolled on trials that achieved their primary radiographic response endpoint versus trials that did not, and for patients who achieved complete or partial response compared to those achieving stable disease.

conclusionsThe prognosis of RRMS enrolled on single-agent phase II trials is poor. This pooled 6-month EFS of RRMS on single-agent trials may be used as a RRMS-specific benchmark for future single-agent phase II trials.

Indexed as

Clinical Trials, Phase II as TopicNeoplasm Recurrence, LocalRhabdomyosarcomaAdolescentChildChild, PreschoolFemaleFollow-Up StudiesHumansInfantMalePrognosisRetrospective StudiesSurvival Rateoncologyphase II clinical trialsrhabdomyosarcoma

Identifiers

PMID38627882
PMCPMC11180298
OpenAlexW4394873832

What OpenQuestion holds

Textmetadata
LicenceTDM
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.