Evidence map›Paper›PMID 28487491›Full record

Trial reportOncotarget2017

A phase II trial of regorafenib in patients with metastatic and/or a unresectable gastrointestinal stromal tumor harboring secondary mutations of exon 17.

Chun-Nan Yeh, Ming-Huang Chen, Yen-Yang Chen, Ching-Yao Yang, Chueh-Chuan Yen, Chin-Yuan Tzen, Li-Tzong Chen, Jen-Shi Chen

Registry-linked trialAbstract readClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Oncotarget, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02606097 (A Phase 2 Study of Regorafenib in Metastatic Gastrointestinal Stromal Tumours With C-KIT exon17 Mutation), which is not on this map. Cited by 37 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
37citing papers in PubMed, 2 pooled it
–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.

NCT02606097 phase2completednot on this map

A Phase 2 Study of Regorafenib in Metastatic Gastrointestinal Stromal Tumours With C-KIT exon17 Mutation

TypeinterventionalSponsorChang Gung Memorial HospitalRan2014 to 2018Enrolled19ConditionsGastrointestinal Stromal Tumour (GIST)Armsregorafenib
3 · Its place in the literature

Who cites it

37 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Guideline
  2. Pooled it
  3. Trial
  4. Trial
  5. Broad spectrum of regorafenib activity on mutant KIT and absence of clonal selection in gastrointestinal stromal tumor (GIST): correlative analysis from the GRID trial.Gastric cancer : official journal of the International Gastric Cancer Association and the Japanese Gastric Cancer Association · 2022
    Trial
  6. Trial
  7. Trial
  8. Trial
  9. Review
  10. Review
  11. Gastrointestinal Stromal Tumors.Current oncology reports · 2025
    Review
  12. Article
  13. Review
  14. Review
  15. Precision Oncology in Gastrointestinal Stromal Tumors.Current oncology (Toronto, Ont.) · 2023
    Review
  16. Review
  17. Review
  18. Article
  19. Review
  20. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

8 authors.

Chun-Nan YehDepartment of Surgery, GIST Team, Chang Gung Memorial Hospital and University, Taoyuan 333, Taiwan.
Ming-Huang ChenSchool of Medicine, National Yang-Ming University, Taipei 112, Taiwan.
Yen-Yang ChenDepartment of Internal Medicine, Division of Hematology-Oncology, Chang Gung Memorial Hospital, Kaohsiung Medical Center, Kaohsiung 833, Taiwan.
Ching-Yao YangDepartment of Surgery, National Taiwan University Hospital, National Taiwan University College of Medicine, Taipei 100, Taiwan.
Chueh-Chuan YenDepartment of Oncology, Taipei Veterans General Hospital, Taipei 112, Taiwan.
Chin-Yuan TzenDepartment of Pathology and Laboratory Medicine, Cathay General Hospital, Taipei 100, Taiwan.
Li-Tzong ChenNational Institute of Cancer Research, National Health Research Institutes, Tainan 70456, Taiwan.
Jen-Shi ChenDivision of Hematology-Oncology, GIST Team, Linkou Chang Gung Memorial Hospital and Chang Gung University, Taoyuan 333, Taiwan.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundGastrointestinal stromal tumors (GISTs) are caused by the constitutive activation of KIT or platelet-derived growth factor receptor alpha (PDGFRA) mutations. Imatinib selectively inhibits KIT and PDGFR, leading to disease control for 80%-90% of patients with metastatic GIST. Imatinib resistance can occur within a median of 2-3 years due to secondary mutations in KIT. According to preclinical studies, both imatinib and sunitinib are ineffective against exon 17 mutations. However, the treatment efficacy of regorafenib for patients with GIST with exon 17 mutations is still unknown. PATIENTS AND

methodsDocumented patients with GIST with exon 17 mutations were enrolled in this study. Patients received 160 mg of oral regorafenib daily on days 1-21 of a 28-day cycle. The primary end point of this trial was the clinical benefit rate (CBR; i.e., complete or partial response [PR], as well as stable disease [SD]) at 16 weeks. The secondary end points of this study included progression free survival (PFS), overall survival, and safety.

resultsBetween June 2014 to May 2016, 18 patients were enrolled (15 of which were eligible for response evaluation). The CBR at 16 weeks was 93.3% (14 of 15; 6 PR and 8 SD). The median PFS was 22.1 months. The most common grade 3 toxicities were hand-and-foot skin reactions (10 of 18; 55.6%), followed by hypertension (5 of 18; 27.8%).

conclusionRegorafenib significantly prolonged PFS in patients with advanced GIST harboring secondary mutations of exon 17. A phase III trial of regorafenib versus placebo is warranted.

trial registrationThis trial is registered at ClinicalTrials.gov in November 2015, number NCT02606097.Key message: This phase II trial was conducted to assess the efficacy and safety of regorafenib in patients with GIST with exon 17 mutations. The results provide strong evidence that regorafenib significantly prolonged PFS in patients with advanced GIST harboring secondary mutations of exon 17.

Indexed as

ExonsMutationAdultAgedAntineoplastic AgentsFemaleGastrointestinal Stromal TumorsHumansKaplan-Meier EstimateMaleMiddle AgedNeoplasm MetastasisNeoplasm StagingPhenylurea CompoundsProto-Oncogene Proteins c-kitPyridinesAntineoplastic AgentsPhenylurea CompoundsProto-Oncogene Proteins c-kitPyridinesregorafenibexon 17GISTregorafenib

Identifiers

PMID28487491
PMCPMC5546467

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Registered trials

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.