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Published on: November 19, 2019
A randomized phase II study of everolimus for advanced pancreatic neuroendocrine tumors in Chinese patients
Jun Yao1, Jian-yao Wang, Yi Liu
1Department of Gastroenterology, Shenzhen Municipal People's Hospital, Jinan University of Medical Sciences, 1017 East Gate Road, Shenzhen, 518020, Guangdong Province, China.
Abstract:
Everolimus, an oral inhibitor of mammalian target of mTOR, has been recently shown to have antitumor effect in a phase III, double-blind, randomized trial (RADIANT-3) of 410 patients with advanced pancreatic neuroendocrine tumors (PNETs). The purpose of this study was to investigate the specific efficacy and safety of everolimus in the Chinese patient with PNETs. In this randomized phase II study, the analysis on Chinese patients was performed comparing efficacy and safety between everolimus 10 mg/day orally (n = 44) and matching placebo (n = 35). The primary endpoint was progression-free survival (PFS). Adverse events were also examined. The median PFS was 15.47 months with everolimus [95% confidence interval (CI) 10.52-26.37], as compared to 4.29 months with placebo (95% CI 2.22-10.75), representing a 72% reduction in the risk of progression or death (hazard ratio 0.27; 95% CI 0.13-0.59; P < 0.001). Drug-associated adverse events (AEs) were mostly grade 1 or 2, observed in all 44 (100%) patients receiving everolimus and in 29 (83%) patients receiving placebo. The most common AEs (grade 1-4) associated with everolimus were rash (n = 38; 86%), stomatitis (n = 30; 68%), infections (n = 33; 75%), epistaxis (n = 32; 73%), pneumonitis (n = 27; 61%) and anemia (n = 22; 50%). Everolimus when compared with placebo is effectively in improving PFS in Chinese patients with PNETs.
Insights
Everolimus significantly improved progression-free survival in Chinese patients with advanced pancreatic neuroendocrine tumors (PNETs). This oral mTOR inhibitor demonstrated a 72% reduced risk of progression or death compared to placebo.
Area of Science:
- Oncology
- Pharmacology
Background:
- Pancreatic neuroendocrine tumors (PNETs) are a rare but significant clinical challenge.
- Everolimus, an oral mTOR inhibitor, has shown prior efficacy in advanced PNETs.
Purpose of the Study:
- To evaluate the efficacy and safety of everolimus in Chinese patients with advanced PNETs.
- To compare progression-free survival (PFS) between everolimus and placebo in this population.
Main Methods:
- Randomized phase II study comparing everolimus (10 mg/day) to placebo in Chinese patients with PNETs.
- Primary endpoint: progression-free survival (PFS).
- Adverse events were systematically recorded and analyzed.
Main Results:
- Median PFS was 15.47 months with everolimus versus 4.29 months with placebo.
- Everolimus demonstrated a 72% reduction in the risk of progression or death (HR 0.27, P < 0.001).
- Most adverse events were grade 1 or 2, with rash, stomatitis, infections, epistaxis, pneumonitis, and anemia being most common with everolimus.
Conclusions:
- Everolimus is effective in improving PFS for Chinese patients with advanced PNETs.
- The safety profile of everolimus in this population is consistent with previous studies, with manageable adverse events.
