Abstract
BackgroundDespite its proven activity as third-line treatment in gastrointestinal stromal tumors (GIST), regorafenib can present a poor tolerability profile which often leads to treatment modifications and transient or permanent discontinuation; thus, in clinical practice physicians usually adopt various dosing and interval schedules to counteract regorafenib-related adverse events and avoid treatment interruption. The aim of this real-world study was to investigate the efficacy and safety of personalized schedules of regorafenib in patients with metastatic GIST, in comparison with the standard schedule (160 mg daily, 3-weeks-on, 1-week-off).Patients and methodsInstitutional registries across seven Italian reference centers were retrospectively reviewed and data of interest retrieved to identify patients with GIST who had received regorafenib from February 2013 to January 2021. The Kaplan–Meier method was used to estimate survival and the log-rank test to make comparisons.ResultsOf a total of 152 patients with GIST, 49 were treated with standard dose, while 103 received personalized schedules. At a median follow-up of 36.5 months, median progression-free survival was 5.6 months [95% confidence interval (CI) 3.73-11.0 months] versus 9.7 months (95% CI 7.9-14.5 months) in the standard-dose and the personalized schedule groups, respectively [hazard ratio (HR) 0.51; 95% CI 0.34-0.75; P = 0.00052]. Median overall survival was 16.6 months (95% CI 14.1-21.8 months) versus 20.5 months (95% CI 15.0-25.4 months), respectively (HR 0.75; 95% CI 0.49-1.22; P = 0.16).ConclusionsRegorafenib-personalized schedules are commonly adopted in daily clinical practice of high-volume GIST expert centers and correlate with significant improvement of therapeutic outcomes. Therefore, regorafenib treatment optimization in patients with GIST may represent the best strategy to maximize long-term therapy.
Highlights
Volume 6 - Issue 4 - 2021 effective therapeutic options were available until the early 2000s, the identification of the role of Proto-Oncogene Tyrosine-Protein Kinase Kit (KIT) and platelet-derived growth factor receptor A (PDGFRA) in the oncogenesis of these malignancies has led to the approval of tyrosinekinase inhibitors, including imatinib, sunitinib, regorafenib, and ripretinib and avapritinib.[3,4]
We aimed to investigate the efficacy and safety of both personalized and standard schedules of regorafenib in patients with metastatic gastrointestinal stromal tumors (GIST), in the real-life Italian clinical setting
A total of 114 (75%) patients were initially treated with regorafenib at the standard dose (160 mg daily, 3-weekson, 1-week-off schedule), while 38 (25%) patients had received personalized treatment upfront for clinical reasons [Eastern Cooperative Oncology Group (ECOG) performance status, age, comorbidities, toxicity to previous treatments]
Summary
Despite its proven activity as third-line treatment in gastrointestinal stromal tumors (GIST), regorafenib can present a poor tolerability profile which often leads to treatment modifications and transient or permanent discontinuation; in clinical practice physicians usually adopt various dosing and interval schedules to counteract regorafenib-related adverse events and avoid treatment interruption. The aim of this real-world study was to investigate the efficacy and safety of personalized schedules of regorafenib in patients with metastatic GIST, in comparison with the standard schedule (160 mg daily, 3-weeks-on, 1-week-off).
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