Efficacy and Long-Term Safety of Everolimus in Pancreatic Neuroendocrine Tumor Associated with Multiple Endocrine Neoplasia Type I: Case Report.

BACKGROUND: Approximately 10% of pancreatic neuroendocrine tumors (NET) are associated with familial syndromes, with the most common type being multiple endocrine neoplasia type 1 (MEN-1). However, the available evidence on how to treat NET comes from studies in sporadic NET. CASE REPORT: Here we report the case of a 51-year-old male patient with a metastatic MEN-1-associated pancreatic NET and hypercalcemia related to primary hyperparathyroidism and tumor-secreted parathyroid-related protein. The patient was treated with everolimus, and showed complete resolution of hypercalcemia and tumor control for 3 years when he presented with pulmonary cryptococcosis and disease progression. CONCLUSION: This case report describes the activity of everolimus in a patient with MEN-1-associated pancreatic NET, its efficacy in treating malignant hypercalcemia associated with NET and the risk of opportunistic infections with prolonged use of this agent.