• Antoine Adenis, Christelle de la Fouchardiere, Bernard Paule, Pascal Burtin, David Tougeron, Jennifer Wallet, Louis-Marie Dourthe, Pierre-Luc Etienne, Laurent Mineur, Stéphanie Clisant, Jean-Marc Phelip, Andrew Kramar, and Thierry Andre.
• Medical Oncology, Centre Oscar Lambret and Catholic University, Lille, France. a-adenis@o-lambret.fr.
• Bmc Cancer. 2016 Jul 7; 16: 412.

BackgroundRandomized trials have shown a survival benefit for regorafenib over placebo in patients with metastatic colorectal cancer (mCRC) that progressed after standard therapies. We evaluated survival and safety outcomes in patients treated with regorafenib in a real-life setting.MethodsREBECCA is a cohort study nested within a compassionate use program designed to evaluate survival, safety, and potential prognostic factors for outcome associated with regorafenib in patients with mCRC refractory to standard therapies. Treatment effects according to various patient and tumour characteristics were evaluated using univariate and multivariate Cox proportional hazards regression models.ResultsOf 1178 patients in the compassionate use program, 654 were in the full analysis set. Median follow-up was 16.5 months. Median survival was 5.6 months. The 12-month survival rate was 22 %. Survival was independently and unfavourably affected by the following variables: poor performance status, short time from initial diagnosis of metastases to the start of regorafenib, low initial regorafenib dose, >3 metastatic sites, presence of liver metastases, and KRAS mutations. We identified prognostic groups of patients with low, intermediate, and high risk of death, with a median survival of 9.2, 5.2, and 2.5 months, respectively. Five-hundred-twenty-four patients (80 %) experienced at least one regorafenib-related adverse event, most commonly, fatigue, hand-foot skin reaction, diarrhea, anorexia, arterial hypertension, and mucositis.ConclusionThe safety and efficacy profile of regorafenib in REBECCA are similar to those in randomized trials. Our prognostic model identified subgroups of mCRC patients who derived a minimal and maximum benefit from regorafenib.Trial RegistrationClinicaltrials.gov NCT02310477 .

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