Treatment options for patients with high-grade pancreatic
neuroendocrine tumors (
pNET) are limited, especially for those with progressive disease and for those who experience treatment failure.
Everolimus, an oral inhibitor of
mammalian target of rapamycin (mTOR), has been approved for the treatment of patients with low- or intermediate-grade advanced
pNET. In the randomized phase III RADIANT-3 study in patients with low- or intermediate-grade advanced
pNET,
everolimus significantly increased progression-free survival (PFS) and decreased the relative risk for
disease progression by 65% over placebo. This case report describes a heavily pretreated patient with high-grade
pNET and liver and peritoneal
metastases who achieved prolonged PFS, clinically relevant partial radiologic
tumor response, and resolution of constitutional symptoms with improvement in Karnofsky performance status while receiving a combination of
everolimus and
octreotide long-acting repeatable (LAR). Radiologic and clinical responses were maintained for 19 months, with minimal toxicity over the course of treatment. This case supports the findings that the combination of
everolimus plus
octreotide LAR may be considered for use in patients with high-grade
pNET and progressive disease. Although behavior and aggressiveness are different between low- or intermediate-grade and high-grade
pNET, some high-grade
pNET may express mTOR; hence,
everolimus should be considered in a clinical trial.