Outcomes
toxicity, death, tumor
DISEASE, EVENTS, TUMOR
The primary objective was to assess the antitumor activity of regorafenib, measured as the rate of assessable patients alive and not progressed after 6 months from study entry. Events were defined as any of the following occurring within 6 months from registration: progression of disease according to RECIST version 1.1, including deterioration of clinical conditions preventing radiological restaging; death for any cause; interruption of regorafenib due to AEs followed by initiation of an alternative antineoplastic treatment.Secondary objectives were to describe toxicity, graded according to CTCAE 4.0; objective response rate (ORR), defined as the number of complete plus partial responses divided by the number of patients enrolled; disease control rate (DCR), defined as the number of complete response, partial response and stable disease, divided by the number of patients enrolled; progression-free survival (PFS), defined as the time from registration to progression or death without progression, whichever occurred first; overall survival (OS), defined as the time from registration to the date of death due to any cause; early metabolic response with [Intra-patient PFS ratios were calculated. Growth modulation index (GMI) was defined as the ratio of the PFS of study treatment with regorafenib to the PFS of the first line of therapy.Translational exploratory objectives aimed to study the molecular determinants of response and resistance to study treatment and included molecular characterization of solid and liquid biopsies in tumor and blood samples. Genetic alterations found in these exploratory studies, as well as their functional characterization, will be reported separately.