A randomized phase II study of everolimus for advanced pancreatic neuroendocrine tumors in Chinese patients.
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.
Department of Gastroenterology, Shenzhen Municipal People's Hospital, Jinan University of Medical Sciences, 1017 East Gate Road, Shenzhen, 518020, Guangdong Province, China.
Jun
J
Yao
Jian-yao
JY
Wang
Yi
Y
Liu
Bin
B
Wang
Ying-xue
YX
Li
Ru
R
Zhang
Li-sheng
LS
Wang
Lei
L
Liu
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D044466:Asian People; D004311:Double-Blind Method; D000068338:Everolimus; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D018358:Neuroendocrine Tumors / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality; D010190:Pancreatic Neoplasms / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality; D020123:Sirolimus / Q000031:analogs & derivatives* / Q000627:therapeutic use; D015996:Survival Rate / Q000639:trends
D000970:Antineoplastic Agents; D007166:Immunosuppressive Agents; D000068338:Everolimus; D020123:Sirolimus