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Updated: Jun 3, 2026
Robotic Duodenal Sleeve Resection for Gastrointestinal Stromal Tumor with Rare Exon 8 KIT Mutation Following Neoadjuvant Imatinib
Published on: April 3, 2026
Phase 2 study of nilotinib as third-line therapy for patients with gastrointestinal stromal tumor
Akira Sawaki1, Toshirou Nishida, Toshihiko Doi
1Department of Gastroenterology, Aichi Cancer Center Hospital, Nagoya, Japan. asawaki@aichi-cc.jp
Background:
Patients with gastrointestinal stromal tumors (GISTs) resistant to both imatinib and sunitinib have a poor prognosis and few therapeutic options. In this study, the efficacy and safety of nilotinib (AMN107) as a third-line therapy for patients with GISTs was evaluated.
Methods:
A single-arm, open-label trial was conducted in 8 Japanese hospitals. The key eligibility criteria included resistance or intolerance to both imatinib and sunitinib treatment. The primary endpoint was disease control rate, defined as the percentage of patients with complete response, partial response (PR), or stable disease (SD) lasting 24 weeks or longer.
Results:
Thirty-five patients were enrolled and treated with nilotinib 400 mg twice daily, which generally was well tolerated. Disease control rate at Week 24 was 29% (90% confidence interval, 16.4%-43.6%). The median progression-free survival was 113 days, and the median overall survival was 310 days. The objective response rate was 3%, comprising 1 PR in a patient with a GIST possessing both a KIT exon 11 mutation, and an imatinib-resistant and sunitinib-resistant KIT exon 17 mutation. Twenty-three (66%) patients had SD (≥6 weeks) as the best response.
Conclusions:
These results suggest that nilotinib is generally well tolerated and has encouraging antitumor activity in patients with GIST who failed both imatinib and sunitinib.
Insights
Nilotinib shows promise as a third-line treatment for gastrointestinal stromal tumors (GISTs) resistant to imatinib and sunitinib. This therapy was generally well-tolerated and demonstrated antitumor activity in heavily pretreated GIST patients.
Area of Science:
- Oncology
- Gastrointestinal Oncology
- Pharmacology
Background:
- Gastrointestinal stromal tumors (GISTs) resistant to imatinib and sunitinib present a significant clinical challenge with limited treatment options.
- Nilotinib (AMN107) is investigated as a potential third-line therapy for these refractory GIST cases.
Purpose of the Study:
- To evaluate the efficacy and safety of nilotinib as a third-line treatment in patients with advanced GIST.
- To determine the disease control rate and survival outcomes in this patient population.
Main Methods:
- An open-label, single-arm clinical trial was conducted across 8 Japanese hospitals.
- Eligibility criteria included documented resistance or intolerance to both imatinib and sunitinib.
- The primary endpoint was the disease control rate at 24 weeks.
Main Results:
- Nilotinib (400 mg BID) was administered to 35 patients and was generally well-tolerated.
- The 24-week disease control rate was 29%, with 66% of patients achieving stable disease.
- Median progression-free survival was 113 days and median overall survival was 310 days.
Conclusions:
- Nilotinib demonstrates encouraging antitumor activity and is generally well-tolerated in patients with GIST who have progressed on imatinib and sunitinib.
- These findings support nilotinib as a viable therapeutic option for refractory GIST cases.
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