Evidence map›Paper›PMID 35950293›Full record

Trial reportCancer medicine2023

A phase II trial of regorafenib in patients with advanced Ewing sarcoma and related tumors of soft tissue and bone: SARC024 trial results.

Steven Attia, Vanessa Bolejack, Kristen N Ganjoo, Suzanne George, Mark Agulnik, Daniel Rushing, Elizabeth T Loggers, Michael B Livingston, Jennifer Wright, Sant P Chawla and 6 more

Open access · goldAbstract readClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Cancer medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 45 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
45citing papers in PubMed, 3 pooled it
5.8field-weighted citation impact, top 3% 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

45 citing papers in PubMed, 3 syntheses or guidelines pooled it, 53 citations in OpenAlex.

  1. Pooled it
  2. Biological Sample Collection to Advance Research and Treatment: A Fight Osteosarcoma Through European Research and Euro Ewing Consortium Statement.Clinical cancer research : an official journal of the American Association for Cancer Research · 2024
    Guideline
  3. Pooled it
  4. Trial
  5. Trial
  6. Trial
  7. Trial
  8. Trial
  9. Randomized Double-Blind Phase II Study of Regorafenib in Patients With Metastatic Osteosarcoma.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2019
    Trial
  10. Observational
  11. Article
  12. Targeted therapies in pediatric oncology: A start.Molecular therapy. Oncology · 2026
    Review
  13. Article
  14. Review
  15. Review
  16. Review
  17. Review
  18. Review
  19. Review
  20. 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

16 authors at 15 institutions in 1 country.

Steven AttiaMayo Clinic Jacksonville, Jacksonville, Florida, USA.
Vanessa BolejackCancer Research and Biostatistics, Seattle, Washington, USA.
Kristen N GanjooStanford Cancer Institute, Stanford, California, USA.
Suzanne GeorgeDana-Farber Cancer Institute/Brigham and Women's Hospital, Boston, Massachusetts, USA.
Mark AgulnikCity of Hope, Duarte, California, USA.
Daniel RushingIndiana University, Bloomington, Indiana, USA.
Elizabeth T LoggersFred Hutchinson Cancer Research Center, Seattle, Washington, USA.
Michael B LivingstonLevine Cancer Institute, Charlotte, North Carolina, USA.
Jennifer WrightHuntsman Cancer Institute, Salt Lake City, Utah, USA.
Sant P ChawlaSarcoma Oncology Center, Santa Monica, California, USA.
Scott H OkunoMayo Clinic, Rochester, Minnesota, USA.
Denise K ReinkeSarcoma Alliance for Research through Collaboration, Ann Arbor, Michigan, USA.ORCID 0000-0002-8662-7060
Richard F RiedelDuke Cancer Institute, Durham, North Carolina, USA.
Lara E DavisOregon Health & Science University, Portland, Oregon, USA.ORCID 0000-0002-6480-1611
Christopher W RyanOregon Health & Science University, Portland, Oregon, USA.
Robert G MakiAbramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.ORCID 0000-0002-9853-2528
Oregon Health & Science University · USBrigham and Women's Hospital · USCancer Prevention Institute of California · USCancer Research And Biostatistics · USCity of Hope · USDuke Cancer InstituteEli Lilly (United States) · USFred Hutch Cancer Center · USIndiana University Bloomington · USJacksonville College · USLevine Cancer Institute · USMayo Clinic · USSarcoma Alliance for Research through Collaboration · USSarcoma Oncology Center · USUniversity of Pennsylvania · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundRegorafenib is one of several FDA-approved cancer therapies targeting multiple tyrosine kinases. However, there are few subtype-specific data regarding kinase inhibitor activity in sarcomas. We report results of a single arm, phase II trial of regorafenib in advanced Ewing family sarcomas.

methodsPatients with metastatic Ewing family sarcomas (age ≥ 18, ECOG 0-2, good organ function) who had received at least one line of therapy and experienced progression within 6 months of registration were eligible. Prior kinase inhibitors were not allowed. The initial dose of regorafenib was 160 mg oral days 1-21 of a 28-day cycle. The primary endpoint was estimating progression-free rate (PFR) at 8 weeks employing RECIST 1.1.

resultsThirty patients (median age, 32 years; 33% women [10 patients]; bone primary, 40%; extraskeletal primary, 60%) enrolled at 14 sites. The most common grade 3 or higher toxicities were hypophosphatemia (5 grade 3, 1 grade 4), hypertension (2 grade 3), elevated ALT (2 grade 3). Sixteen patients required dose reductions, most often for hypophosphatemia (n = 7 reductions in 6 patients); two stopped regorafenib for toxicity. There was one death unrelated to treatment in the 30-day post-study period. Median progression-free survival (PFS) was 14.8 weeks (95% CI 7.3-15.9); PFR at 8 weeks by Kaplan-Meier analysis was 63% (95% CI 46-81%). The RECIST 1.1 response rate was 10%. Median OS was 53 weeks (95% CI 37-106 weeks).

conclusionsRegorafenib has modest activity in the Ewing family sarcomas. Toxicity was similar to that seen in approval studies.

Indexed as

Bone NeoplasmsHypophosphatemiaSarcomaSarcoma, EwingAdultFemaleHumansInfantMalePhenylurea CompoundsPyridinesPhenylurea CompoundsPyridinesregorafenib(5): CIC-DUX4clinical trialEwing sarcomaregorafenib

Identifiers

PMID35950293
PMCPMC9883574
OpenAlexW4292712479

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.