Introduction: Everolimus is an oral mTOR inhibitor that exerts antineoplastic effects inhibiting cell proliferation, survival and angiogenesis. Its activity in advanced neuroendocrine tumors (NETs) has been demonstrated in controlled trials and everolimus was approved by the FDA for the treatment of progressive, advanced pNETs in May 2011.
Materials and methods: We treated with everolimus, at the dosage of 10 mg once daily, 14 patients with advanced, progressive, low- or intermediate-grade NETs for a mean period of 11 months. Somatostatin analogues treatment was continued in all patients. Twelve of 14 patients had previously undergone peptide receptor radionuclide therapy (PRRT) with either Lutetium or Yttrium.
Results: According to RECIST criteria, stable disease was observed in 9/14 patients and partial response was achieved in 2/14 patients. Median progression-free survival was 12.0 months. Drug-related adverse events included stomatitis (7/14), hyperglycaemia (7/14), hypertriglyceridemia (5/14), pneumonitis (4/14), hematologic toxicity (4/14), peripheral oedema (4/14) and rash (2/14). Grade 3 and 4 adverse events included pneumonitis (three cases) and thrombocytopenia (two cases). Dose reduction was required in 5/14 patients.
Conclusion: Our data confirm the efficacy of everolimus in the treatment of progressive, advanced NETs. The apparently higher rate of grade 3 and 4 adverse events is probably related to the high proportion of patients in our series that had previously undergone PRRT, as it may enhance everolimus potential myelotoxicity.
27 Apr - 01 May 2013
European Society of Endocrinology