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Updated: Aug 29, 2026
Molecular and Immunologic Techniques in a Genetically Engineered Mouse Model of Gastrointestinal Stromal Tumor
Published on: May 2, 2022
[Effect of imatinib treatment of gastrointestinal stromal tumors]
Sándor Eckhardt1, Zsuzsanna Pápai, György Bodoky
1Országos Onkológiai Intézet, B Belgyógyászati Osztály, Budapest. eckhardt@oncol.hu
Introduction:
Advanced malignant gastrointestinal stromal tumours are practically resistant to further radio- or chemotherapy. These tumours are characterized by the presence of C-KIT (a transmembrane tyrosin kinase) mutation which can be specified by CD117 expression. Imatinib (2-fenilaminopirimidine) is a selective inhibitor of the mutated C-KIT.
Aim:
The purpose of our study was to determine the potential antitumour effect of imatinib in patients with gastrointestinal stroma tumour patients.
Materials And Methods:
An open, non-randomized trial was performed involving 38 patients each of which had received/metastatic disease associated with CD117 positivity. Consecutively daily doses of 400-600 mg imatinib was administered orally to the patients. The evaluation was carried out on 37 patients in a form of an interim analysis.
Results:
After a 3-18 months observation period 1 complete, 19 partial remissions and 10 static diseases could be registered (78%), in association of only grade 1-2 toxicity.
Conclusions:
The imatinib treatment improved the quality of life of the patients with gastrointestinal stroma tumour and their life expectancy became considerably prolonged. Further follow-up of the patients as well as design of a prospective, randomized trial on a larger patient material is urgently needed.
Insights
Imatinib effectively treats advanced gastrointestinal stromal tumors (GIST) by targeting C-KIT mutations. This targeted therapy shows significant patient response rates and improved life expectancy with manageable toxicity.
Area of Science:
- Oncology
- Pharmacology
Background:
- Advanced gastrointestinal stromal tumors (GIST) exhibit resistance to conventional radio- and chemotherapy.
- These tumors are characterized by C-KIT (CD117) mutations, a target for specific therapies.
- Imatinib is a selective inhibitor of the mutated C-KIT tyrosine kinase.
Purpose of the Study:
- To evaluate the antitumour efficacy of imatinib in patients diagnosed with gastrointestinal stromal tumors.
- To assess the impact of imatinib on patient outcomes in advanced GIST.
Main Methods:
- An open-label, non-randomized trial was conducted with 38 patients presenting metastatic, CD117-positive GIST.
- Patients received daily oral doses of imatinib ranging from 400-600 mg.
- Interim analysis included 37 patients after a 3-18 month observation period.
Main Results:
- A significant overall response rate of 78% was observed, including 1 complete remission and 19 partial remissions.
- 10 patients achieved static disease, indicating tumor stabilization.
- Treatment was associated with only grade 1-2 toxicity, suggesting a favorable safety profile.
Conclusions:
- Imatinib treatment demonstrably improves the quality of life and prolongs life expectancy for patients with advanced GIST.
- Further investigation through prospective, randomized trials with larger patient cohorts is warranted.
- Imatinib represents a promising therapeutic option for C-KIT-driven gastrointestinal stromal tumors.
