Abstract 116P
Background
Ripretinib, a switch-control tyrosine kinase inhibitor designed to broadly inhibit KIT and PDGFRA kinase signaling, has been approved for treatment of advanced gastrointestinal stromal tumors (GISTs). Given its promising efficacy and safety profile, the administration of ripretinib as a preoperative therapy presents a strategic approach to downstage the GISTs that are initially unresectable, recurrent or metastatic, thus potentially facilitating subsequent surgical intervention. This study aims to investigate the efficacy of ripretinib in potentially resectable metastatic GIST following failure of imatinib therapy.
Methods
This is a single-arm, multi-center exploratory study (NCT05132738). Key eligibility criteria include potentially resectable locally advanced or recurrent metastatic GIST who have failed imatinib treatment: 1) the number of evaluable lesions is ≤5; 2) resection is considered to have significant risks by MDT assessment (meeting any of the following): a) the maximum diameter of a single lesion is ≥10 cm; b) organ function damage surgery is required; c) multiple organ resection surgery is required. Patients were orally administered 150 mg once daily (qd) of ripretinib for up to six treatment cycles before surgical intervention. The primary endpoint focused on the no evidence of disease (NED) rate, while secondary endpoints are the R0/R1 resection rate, surgery rate, and objective response rate.
Results
As of July 12, 2024, a total of 12 patients were enrolled in the study. 2/12 (16.7%) patients experienced partial response as the best response and a further 7/12 (58.3%) patients maintained stable disease. Surgical resection was successfully performed in 4/12 (33.3%) patients, achieving the primary endpoint without any surgical complications. Furthermore, no treatment-related adverse events led to drug discontinuation or death.
Conclusions
Ripretinib may enhance surgical outcomes in potentially resectable GISTs after the failure of imatinib therapy, demonstrating a clinical benefit with a 33.3% NED rate.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
The authors.
Funding
Has not received any funding.
Disclosure
All authors have declared no conflicts of interest.