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Safety of Imatinib Mesylate in a Multicenter Expanded Access Program in Adult Patients with Gastrointestinal Stromal
Peter Reichardt1, Marcus Schlemmer2, Juan R Delgado Perez3
1Department of Oncology and Palliative Care, Sarcoma Center Berlin-Brandenburg, Helios Klinikum Berlin-Buch, Berlin, Germany.
Background:
Gastrointestinal stromal tumors (GISTs) are mesenchymal tumors most often caused by activating mutations of the KIT gene. KIT tyrosine kinase inhibitors provide targeted therapy for the underlying genetic mutation, and adjuvant therapy is indicated for patients who are at significant risk of relapse following GIST resection. This is a report of the safety of imatinib in patients with GIST in the adjuvant setting in an expanded access program.
Methods:
In this multicenter, open-label, single-arm trial, safety was assessed based on the frequency of adverse events (AEs).
Results:
Three hundred patients were treated and analyzed; 40 patients discontinued treatment. Median overall exposure during the program was 181 days (range 9-420); most patients (260/300 treated) completed the study. Six patients had disease recurrence, 4 of whom discontinued. In line with previously published reports, the most frequent AEs were nausea, diarrhea, and periorbital edema. The AEs were mild to moderate in most cases (76%).
Conclusions:
These findings are in agreement with the known safety profile of imatinib and confirm the safety of imatinib at 400 mg/day in the adjuvant setting. The incidence of severe AEs was low.
Insights
This study confirms imatinib is safe for adjuvant therapy in gastrointestinal stromal tumor (GIST) patients. Most adverse events were mild to moderate, with a low incidence of severe side effects.
Area of Science:
- Oncology
- Gastrointestinal Oncology
- Pharmacology
Background:
- Gastrointestinal stromal tumors (GISTs) are mesenchymal neoplasms often driven by KIT gene mutations.
- KIT tyrosine kinase inhibitors offer targeted therapy for GIST.
- Adjuvant therapy is crucial for high-risk GIST patients post-resection.
Purpose of the Study:
- To evaluate the safety and tolerability of imatinib in the adjuvant setting for GIST patients.
- To assess adverse events (AEs) in a large cohort undergoing adjuvant imatinib therapy.
Main Methods:
- Multicenter, open-label, single-arm trial design.
- Safety assessment based on the frequency and severity of adverse events.
- Analysis of 300 patients treated in an expanded access program.
Main Results:
- Most patients (260/300) completed the study with a median exposure of 181 days.
- Common AEs included nausea, diarrhea, and periorbital edema, mostly mild to moderate (76%).
- Six patients experienced disease recurrence; 4 discontinued treatment due to recurrence.
Conclusions:
- Imatinib at 400 mg/day demonstrates a consistent safety profile in the adjuvant GIST setting.
- The incidence of severe adverse events was notably low.
- Findings support the use of imatinib for adjuvant therapy in GIST patients at risk of relapse.
