Regorafenib treatment for advanced, refractory gastrointestinal stromal tumor: A report of the U.K. Managed Access Program.

2014 
10551 Background: Tyrosine kinase inhibitors (TKI) have revolutionized the treatment of gastrointestinal stromal tumours (GIST) although most patients develop resistance to first and second-line therapies. Regorafenib, an oral multi-targeted TKI, has demonstrated benefit in previously treated GIST patients. Methods: We assessed safety and preliminary activity of regorafenib in patients treated within the Managed Access Programme (MAP). All consecutive patients with advanced GIST who had progressed on or were intolerant of imatinib and sunitinib were recruited from the Royal Marsden Hospital and University College Hospital. We retrospectively reviewed the data for response, toxicity and treatment duration. Response was assessed by RECIST and Choi criteria. Toxicity was graded according to CTCAE v4.0 criteria. Results: 20 patients were included in the MAP in the UK between 3/2013 and 9/2013. Median age was 68 (range 45-87), 65% of patients were male. Performance Status was 0-1 for 18 patients (90%), 2 for 2...
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