Long-term everolimus treatment of patients with pancreatic neuroendocrine tumors

Background/Aims: Based on the significant prolongation of progression-free survival in a randomized phase III trial, RADIANT-3 (RAD001 in Advanced Neuroendocrine Tumors, Third Trial), everolimus has been approved for the management of advanced, progressive pancreatic neuroendocrine tumors (pNET). Here, we describe 15 participants in RADIANT-3 who were treated with everolimus at our study center. We report the long-term survival of a subset of patients. Methods: Patients with advanced, progressive pNET were randomly assigned to the everolimus arm of RADIANT-3 or received everolimus as open-label treatment after experiencing progression on placebo or during the unblinded phase. Results: Five patients on everolimus (5-10 mg/day) had stable disease for >43 to >76 months after initiating treatment. Three patients achieved stable disease for 19-25 months, but died of progressive malignancy thereafter. Seven patients had stable disease for ≤11 months after initiating everolimus therapy. Conclusion: Patients with advanced, progressive pNET can obtain long-term benefit from daily oral treatment with everolimus.