Impact of everolimus on Japanese patients with advanced pancreatic neuroendocrine neoplasms

Background Although everolimus has become a key therapeutic agent in patients with advanced pancreatic neuroendocrine neoplasms ( PNEN ), its efficacy and safety in clinical practice remains unclear. Methods Forty‐seven patients with advanced PNEN treated with everolimus were reviewed retrospectively. To evaluate the safety of everolimus as a long‐term treatment, the patients were divided into two groups according to treatment duration: group A, ≤1 year ( n  =   21); group B, >1 year ( n  =   26). Results Among 42 patients with pancreatic neuroendocrine tumors ( PNET ), the median progression‐free survival, overall survival, and objective response rate were 27.5 months, 60.8 months, and 19.0%, respectively. Two patients with pancreatic neuroendocrine carcinomas ( PNEC ) with lower Ki‐67 index and well‐differentiated tumors showed favorable responses. More patients in group A discontinued everolimus owing to adverse drug reactions ( ADR s) than in group B. The median relative dose intensity was significantly lower in group B than group A ( P  =   0.045), whereas the drug interruption rate was significantly higher in group B than group A ( P  <   0.001). Conclusions Everolimus showed significant clinical benefit in Japanese patients with advanced PNEN . Prevention of severe ADR s by appropriate dose reduction and interruption is necessary for a long‐term continuation of everolimus.