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Preoperative Avapritinib for Localized PDGFRA-Mutant GIST: Marked Pathologic Response, but Limited Feasibility at
Tannaz Ranjbarian1,2, Mark Antkowiak1,2, Shirley Sarno1,2
1Division of Surgical Oncology, Department of Surgery, University of California San Diego, San Diego, California, USA.
Cancer Medicine
|July 30, 2026
Summary
Preoperative avapritinib showed significant tumor reduction in localized PDGFRA-mutant GIST, but frequent toxicities limited its use. Further trials are needed to define its role in GIST treatment.
Area of Science:
- Oncology
- Gastrointestinal Oncology
- Precision Medicine
Background:
- Avapritinib is approved for advanced PDGFRA exon 18-mutant GIST.
- Its preoperative use in localized GIST is not well-defined.
Purpose of the Study:
- To evaluate the efficacy and safety of preoperative avapritinib in localized, resectable PDGFRA-mutant GIST.
- To assess radiographic and pathologic responses, treatment duration, adverse events, and surgical outcomes.
Main Methods:
- Retrospective, two-center case series of eight patients.
- Patients received preoperative avapritinib (300 mg daily).
- Evaluated radiographic/pathologic response, AEs, and surgical outcomes.
Main Results:
- Tumor size decreased in 88% of patients (median 25% reduction).
- 50% experienced Grade ≥3 AEs; dose reductions were needed in 38%.
- 5 patients underwent resection with complete or near-complete pathologic response.
Conclusions:
- Preoperative avapritinib demonstrated pathologic activity in localized PDGFRA-mutant GIST.
- Frequent early toxicity at the standard dose limited surgical access for some.
- Routine preoperative use is not recommended; consider for trials or selected patients.
