Trial reportClinical cancer research : an official journal of the American Association for Cancer Research2022
Phase II Study of Ponatinib in Advanced Gastrointestinal Stromal Tumors: Efficacy, Safety, and Impact of Liquid Biopsy and Other Biomarkers.
Trial report in Clinical cancer research : an official journal of the American Association for Cancer Research, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 1 of them a synthesis that pooled 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.
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.
Who cites it
19 citing papers in PubMed, 1 synthesis or guideline pooled it, 24 citations in OpenAlex.
- Prognostic Significance of Circulating Tumor DNA Mutations in Gastrointestinal Stromal Tumors: A Systematic Review and Meta-analysis Based on Time-To-Event Data.Journal of gastrointestinal cancer · 2025Pooled it
- Precision therapeutic strategies for advanced gastrointestinal stromal tumors.Cancer metastasis reviews · 2026Review
- Interpreting ctDNA dynamics in gastrointestinal stromal tumor (GIST) therapy-timing may be key.Journal of gastrointestinal oncology · 2026Article
- Emerging mechanisms of kinase inhibitor escape and clinical implications of ponatinib in advanced gastrointestinal stromal tumor.Journal of gastrointestinal oncology · 2026Article
- Liquid biopsies in precision oncology for older adults with cancer.NPJ precision oncology · 2026Review
- Molecular Mechanisms and Clinical Implications ofCurrent issues in molecular biology · 2025Review
- Tyrosine Kinase Inhibitors for Gastrointestinal Stromal Tumor After Imatinib Resistance.Pharmaceutics · 2025Review
- Alleviation of Neurological Disorders by Targeting Neurodegenerative-Associated Enzymes: Natural and Synthetic Molecules.International journal of molecular sciences · 2025Review
- Clinical Use of Liquid-Based Comprehensive Genomic Profiling in Gastrointestinal Stromal Tumors.Laboratory investigation; a journal of technical methods and pathology · 2025Article
- Recent Advancement in Drug Designing as Small Molecules in Targeted Cancer Therapy: Challenges and Future Directions.Current cancer drug targets · 2025Review
- Gastrointestinal Stromal Tumors (GISTs) in Pediatric Patients: A Case Report and Literature Review.Children (Basel, Switzerland) · 2024Review
- Pilot Study by Liquid Biopsy in Gastrointestinal Stromal Tumors: Analysis ofInternational journal of molecular sciences · 2024Article
- Pan-Canadian consensus recommendations for GIST management in high- and low-throughput centres across Canada.Therapeutic advances in medical oncology · 2024Review
- Review
- Review
- Small molecule inhibitors targeting the cancers.MedComm · 2022Review
- Review
- Gastrointestinal Stromal Tumors: What Is the Best Sequence of TKIs?Current treatment options in oncology · 2022Review
- The GIST of Advances in Treatment of Advanced Gastrointestinal Stromal Tumor.American Society of Clinical Oncology educational book. American Society of Clinical Oncology. Annual Meeting · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 5 institutions in 2 countries.
Funding
Abstract
purposeThe purpose of this study is to evaluate ponatinib for advanced gastrointestinal stromal tumors (GIST). PATIENTS AND
methodsThis single-arm phase II trial enrolled patients with metastatic and/or unresectable GIST with failure of prior tyrosine kinase inhibitor (TKI) treatment into two cohorts based on presence or absence of KIT exon 11 (ex11) primary mutations. Patients initially received ponatinib 45 mg once daily. Following a temporary clinical hold in October 2013, dose reductions were implemented to reduce risk of arterial occlusive events (AOE). Primary endpoint was 16-week clinical benefit rate (CBR) in KIT ex11-positive cohort. KIT mutations in circulating tumor DNA (ctDNA) were assessed.
resultsForty-five patients enrolled (30 KIT ex11-positive and 15 KIT ex11-negative); median follow-up was 14.7 and 13.6 months, respectively, as of August 1, 2016. Sixteen-week CBR was 36% (KIT ex11-positive; primary endpoint) and 20% (KIT ex11-negative). ctDNA analyses (n = 37) demonstrated strong concordance of primary KIT mutations between plasma and tumor. At least two secondary mutations were detected in 35% of patients overall and 54% of KIT ex11-positive patients. Changes from baseline in mutated ctDNA levels were consistent with clinical activity. Ponatinib was ineffective in patients with KIT exon 9 primary mutations. Resistance was associated with emergence of V654A. AOEs and venous thromboembolic events occurred in three and two patients, respectively. Six patients died; two deaths (pneumonia and pulmonary embolism) were considered possibly ponatinib-related.
conclusionsPonatinib demonstrated activity in advanced GIST, particularly in KIT ex11-positive disease. ctDNA analysis confirmed heterogeneous resistance mutations in TKI-pretreated advanced GIST. Safety was consistent with previous studies.
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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.