Efficacy and Safety of Everolimus in Metastatic Gastroenteropancreatic Neuroendocrine Tumors.

Introduction: Everolimus is a treatment option for patients with pancreatic NETs, while its use in patients with gastrointestinal NETs still remains to be defined. Aim(s): To test the efficacy and side effects of Everolimus in six patients with differentiated NET refractory to other therapies. Materials and methods: Everolimus (10 mg/day; compassionate use) was administered until disease progression or unacceptable toxicity. Three patients had metastases of ileal neuroendocrine carcinoma; one of typical lung carcinoid; one of ovary carcinoid and one of pancreatic insulinoma. Results: Two patients showed stable disease at imaging after six and 12 months of treatment. One patient showed biochemical remission. Two patients discontinued treatment due to adverse events. One patient did not reach the six months followup. Side effects were: stomatitis (2); thrombocytopenia (2); nausea (2); fatigue (2) that in one case determined definitive therapy discontinuation after eight months; skin infected lesions (1) leading to definitive discontinuation after three months of therapy. In one case, Everolimus was discontinued after 17 months for severe tricuspid valve insufficiency. Finally, one patient discontinued therapy after three months for worsening dyspnea. The only patient currently under treatment had no side effects. During therapy, lipid and glucose profile did not show significant alterations. Conclusion: Everolimus induces disease stabilization but may have several side effects which should be monitored. [ABSTRACT FROM AUTHOR]