Clinical outcomes and prognostic factors for patients with high-risk gastrointestinal stromal tumors treated with 3-year adjuvant imatinib

Three years of adjuvant imatinib is the current standard for patients with high-risk gastrointestinal stromal tumors (GIST). We aimed to investigate the safety and efficacy profiles of 3-year imatinib, focusing on the prognostic value of various factors. In this registry-based study, 222 patients with high-risk GIST who underwent surgical resection followed by 3 years of adjuvant imatinib between 2010 and 2018 were included. The imatinib dose was reduced in 39 (17.6%), and 13 (5.9%) discontinued imatinib due to toxicity. With a median follow-up duration of 65.7 months, 5-year recurrence-free survival (RFS) and overall survival (OS) were 73.2% and 93.9%, respectively. Tumor rupture, tumor size of >10 cm, mitotic index of >10/50 high power fields (HPF) were independent factors for short RFS. Patient subgroups stratified by the risk factors showed distinct RFS (P < .001): patients without the above risk factors or those with only a tumor size of >10 cm showed favorable RFS (5-year RFS 83.8% and 92.3%, respectively), whereas those with tumor rupture or those with tumor size of >10 cm and mitotic index of >10/50 HPF showed prominently poor RFS (5-year RFS of 54.8% and 47.9%, respectively). Three years of adjuvant imatinib treatment was generally tolerable and effective, which were consistent with the clinical outcomes of previous reports. The presence of tumor rupture, large tumor and high mitotic count was independently associated with poor RFS. Based on these risk factors, different management strategies, such as different durations of adjuvant imatinib, deserve further investigation.