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Imatinib in c-KIT-mutated metastatic solid tumors: A multicenter trial of Korean Cancer Study Group (UN18-05 Trial)
Hye Ryeon Kim1,2, Su Jin Lee3, Mi Sun Ahn4
1Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
Introduction:
We conducted an open-label, single-arm, multi-center phase II trial to evaluate the efficacy and safety of imatinib chemotherapy-refractory or metastatic solid tumor patients with c-KIT mutations and/or amplification.
Methods:
c-KIT mutations and amplification were detected using NGS. Imatinib (400 mg daily) was administered continuously in 28-day cycles until disease progression, unacceptable adverse events, or death by any cause. The primary endpoint was the objective response rate (ORR).
Result:
In total, 18 patients were enrolled on this trial. The most common tumor type was melanoma (n = 15, 83.3%), followed by ovarian cancer, breast cancer, and metastasis of unknown origin (MUO) (each n = 1, 5.5%). The total number of evaluable patients was 17, of which one patient had a complete response, six patients had partial response, and two patients had stable disease. The overall response rate (ORR) of 41.2% (95% CI 17.80-64.60) and a disease control rate of 52.9% (95% CI 29.17-76.63). The median progression-free survival was 2.2 months (95% CI 1.29-3.20), and median overall survival was 9.1 months (95% CI 2.10-16.11). The most common adverse events were edema (31.3%), anorexia (25.0%), nausea (18.8%), and skin rash (18.8%).
Conclusion:
Imatinib demonstrated modest anti-tumor activity and a manageable safety profile in chemotherapy-refractory solid tumors with c-KIT mutation, especially in melanoma patients.
Insights
Imatinib showed modest effectiveness in treating solid tumors with c-KIT mutations, particularly in melanoma patients. The treatment offered a manageable safety profile for those refractory to chemotherapy.
Area of Science:
- Oncology
- Medical Genetics
- Clinical Pharmacology
Background:
- Investigated imatinib efficacy in patients with chemotherapy-refractory solid tumors harboring c-KIT mutations or amplification.
- Utilized an open-label, single-arm, multi-center Phase II clinical trial design.
Purpose of the Study:
- To evaluate the efficacy and safety of imatinib in a specific patient population.
- To determine the objective response rate (ORR) as the primary endpoint.
Main Methods:
- Detected c-KIT mutations and amplification using next-generation sequencing (NGS).
- Administered imatinib 400 mg daily in 28-day cycles until disease progression, adverse events, or death.
- Monitored patients for response, survival, and adverse events.
Main Results:
- 18 patients enrolled; 17 were evaluable. Melanoma was the most common tumor type (83.3%).
- Achieved an overall response rate (ORR) of 41.2% and a disease control rate of 52.9%.
- Median progression-free survival was 2.2 months; median overall survival was 9.1 months. Common adverse events included edema and anorexia.
Conclusions:
- Imatinib demonstrated modest anti-tumor activity in chemotherapy-refractory solid tumors with c-KIT mutations.
- The safety profile of imatinib was manageable in this patient cohort.
- Melanoma patients with c-KIT mutations showed particular benefit from imatinib treatment.
