Neoadjuvant tyrosine kinase inhibitor therapy for patients with gastrointestinal stromal tumor: A propensity-matched analysis

Background: Tyrosine kinase inhibitor (TKI) neoadjuvant therapy (NAT) is often given in gastrointestinal stromal tumors (GISTs) with the goal to facilitate less morbid resections and improve oncologic outcomes; however, the use of NAT for GIST is poorly studied. Methods: We reviewed patients with resected nonmetastatic GIST from 2003 to 2019. Overall (OS) and recurrence-free survival (RFS) were assessed with Kaplan-Meier modeling. We performed 1:1 propensity-matching for relevant clinicopathologic variables for receipt of NAT. Results: We identified 254 patients. Propensity 1:1 matching resulted in 33 patients per group. The median follow-up was 77 months with no difference in 10-year OS (68% vs. 73%), 5-year RFS (13% vs. 10%), or median RFS (24 vs. 27 months) for patients treated with NAT versus upfront resection (all P > 0.9). Hospital length-of-stay (both median 7 days) and Clavien-Dindo ≥ III complications (12% vs. 3%) were not different between groups (both P ≥ 0.35). Discussion: TKI NAT can be used to facilitate resection in select patients with surgically higher-risk GIST, however it does not result in an independent oncologic benefit.