Progression-free survival improved in patients with advanced neuroendocrine tumors associated with carcinoid syndrome when the patients used everolimus rather than placebo in combination with octreotide long-acting repeatable (LAR).

Neuroendocrine tumors, also known as carcinoids, are uncommon tumors that arise from various primary sites and frequently spread to the liver, causing various symptoms referred to as carcinoid syndrome. Currently, no drug is FDA-approved for the oncologic control of most neuroendocrine tumors. Everolimus, an oral inhibitor of the mammalian target of rapamycine (mTOR), has shown antitumor activity in persons with advanced pancreatic neuroendocrine tumors.

As reported in The Lancet (2011;378[9808]:2005-2012), 71 persons 18 years and older completed the phase 3 RADIANT-2 study in which they were randomized to 10 mg oral everolimus or placebo per day, both in conjunction with 30 mg intramuscular octreotide LAR every 28 days. All participants had low-grade or intermediate-grade advanced (unresectable locally advanced or distant metastatic) neuroendocrine tumors.

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Median progression-free survival was 16.4 months in the everolimus group and 11.3 months for the placebo users. Drug-related adverse events were higher but manageable in the everolimus arm, mostly grade 1 or 2. Adverse events of all grades included stomatitis in 62% of the everolimus patients and 14% of the placebo group; rash (37% and 12%, respectively), fatigue (31% and 23%, respectively), and diarrhea (27% and 16%, respectively).