Sun W, et al. - In this single arm, phase 2 study, regorafenib was tested as treatment option in patients who had advanced/unresectable or metastatic disease after receiving standard therapy. Regorafenib was administered to patients once daily on a schedule of 21-days on/7-days off in a 28-day cycle. A standard 160 mg dose was initially administered. Toxicity assessments in the first 3 patients were followed by dose reduction to 120 mg for subsequent patients. Promising efficacy of regorafenib was seen, with median progression-free survival (PFS) 15.6 weeks and median overall survival (OS) 31.8 weeks, and survival rates 40% at 12 months and 32% at 18 months. Five patients (11%) achieved a partial response, and stable disease (44%) was reported in 19, for a disease control rate of 56%. In 40% of patients, grade 3 and 4 adverse events occurred, with most common toxicities being hypophosphatemia (40%), hyperbilirubinemia (26%), hypertension (23%), and hand-foot skin reaction (7%).