Everolimus in patients with advanced follicular-derived thyroid cancer; results of a phase II clinical trial.

Background:mTOR upregulation has been reported to be involved in the pathogenesis of thyroid tumors and treatment with the mTOR inhibitor everolimus has shown promising results in endocrine tumors. We conducted a prospective phase II clinical trial to determine the efficacy and safety of everolimus in patients with advanced differentiated thyroid cancer.Patients and methods:Twenty-eight patients with progressive metastatic or locally advanced radioactive refractory differentiated thyroid cancer and 7 patients with anaplastic thyroid cancer were included and received everolimus 10mg orally once daily. The primary endpoint was disease control rate (complete (CR) + partial response (PR) + stable disease (SD) >24 weeks). Secondary endpoints included progression free survival (PFS), overall survival (OS), toxicity, mutational related outcomes and pharmacokinetic related outcomes (PK).Results:Median follow-up duration was 38 months (2-64). Seventeen patients (65%) showed SD, of which 15 (58%) showed SD > 24 wee...