Rare Disease Report

Everolimus Slows Neuroendocrine Tumor Progression

OCTOBER 19, 2015
Steve Carrell
Everolimus produced 7.1 months of progression-free survival in patients with lung/gastrointestinal neuroendocrine tumors (NETS) showing a 52% risk reduction in progression.
"RADIANT-4 showed everolimus is the first targeted agent to show robust anti-tumor activity, with acceptable tolerability, against a broad spectrum of NET , including those arising from the lung and GI tract," said James C Yao, MD, professor of medical oncology at the University of Texas M.D. Anderson Cancer Center. According to Yao, RADIANT-4 produced unequivocal evidence of efficacy with the tumors studied. It was the first large, placebo controlled, Phase III study of advanced, progressive, nonfunctional lung/GI NET.
According to Yao, benefits appeared and were durable at 18 months. Death rates in the everolimus and placebo group were similar, although the everolimus group was treated longer, Yao said. He added that the next interim analysis will occur in 2016.
RADIANT-4 was the first randomized study to show everolimus produced significant results with lung tumors in 31% of everolimus patients. Tumor shrinkage of any degree occurred in 64% of the everolimus group and 26% of the placebo group.
RADIANT-4's full analysis set had 97 placebo patients and 205 patients taking 10 mg a day of everolimus for a median of 40.4 weeks. Tumor origins were gastrointestinal, 175 patients; lung, 90; unknown origin, 36. Disease progression occurred in 37% of everolimus patients and 72% of placebo patients. Adverse events accounted for discontinuations in 29% and 7% of the groups, respectively, Yao said.

About Neuroendocrine Tumors

Neuroendocrine tumors (NETS) comprise rare neoplasms that are derived from neuroendocrine cells interspersed within the body – often from the gastrointestinal system.
Neuroendocrine tumors produce various hormonal syndromes and for their indolent clinical course in most patients, although some of these tumors do not produce hormones of clinical significance. Patients may have symptoms for many years before the diagnosis is suspected and confirmed. Therefore, the index of suspicion must be high in order to diagnose these tumors in a timely fashion.
Symptoms vary greatly with each patient and management of NETS is often a multidisciplinary approach.

Image by Nephron courtesy of Wikimedia Commons

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