title: Safety and QOL in Patients with Advanced NET in a Phase 3b Expanded Access Study of Everolimus.
abstract: An open-label, multi-center, expanded access study was conducted in patients with advanced neuroendocrine tumors (NET) treated with everolimus (10 mg/day) to assess safety and health-related quality of life (HRQOL).
  
  
  
  Of the 246 patients enrolled, 126 have pancreatic NET (pNET) and 120 have non-pNET. Patients continued treatment until disease progression, unacceptable toxicity, death, until commercial availability of everolimus, or May 2012, whichever came first. Adverse events (AEs) were analyzed according to Common Terminology Criteria version 4.0. HRQOL was assessed at baseline, for three 28-day cycles, and then at every three cycles until end of treatment (EOT) with EQ-5D, EORTC QLQ-C30, and EORTC QLQ-GINET21 instruments.
  
  
  
  The most common grade 3 or 4 AEs included hyperglycemia, infections, stomatitis, fatigue, and abdominal pain. In patients with pNET, mean (± SD) EQ VAS score remained stable at EOT (baseline, 68.8 ± 19.9 vs. EOT, 66.5 ± 20.6) without clinically significant change in QLQ-C30 global health status (change from baseline, - 3.9; n = 86). For patients with non-pNET, a reduction in EQ VAS score (63.9 ± 19.0 vs. 55.3 ± 23.0) with clinically significant changes in QLQ-C30 global health status (-13.0; n = 69) was seen by EOT. EQ-5D utility scores remained stable in patients with pNET and a moderate decrease was reported by patients with non-pNET.
  
  
  
  The safety profile of everolimus was consistent with the previous studies without adversely affecting HRQOL in pNET. Lower baseline HRQOL scores and more frequent comorbidities might have contributed to the worse outcomes in non-pNET.
  
  
  
  EudraCT no. 2010-023032-17.
authors.affiliation: Department of Hepatology and Gastroenterology, Charité Universitätsmedizin Berlin, Campus Virchow Klinikum, Berlin, Germany. Marianne.Pavel@charite.de.
authors.forename: Marianne
authors.initials: M
authors.lastname: Pavel
authors.affiliation: University of Duisburg-Essen, Essen, Germany.
authors.forename: Nicole
authors.initials: N
authors.lastname: Unger
authors.affiliation: Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels, Belgium.
authors.forename: Ivan
authors.initials: I
authors.lastname: Borbath
authors.affiliation: Presidio Ospedaliero Santa Chiara, Pisa, Italy.
authors.forename: Sergio
authors.initials: S
authors.lastname: Ricci
authors.affiliation: Chang Gung Memorial Hospital, Chang Gung University, Lin-Kou, Taiwan.
authors.forename: Tsann-Long
authors.initials: TL
authors.lastname: Hwang
authors.affiliation: Novartis Pharma S.A.S., Rueil-Malmaison, France.
authors.forename: Thomas
authors.initials: T
authors.lastname: Brechenmacher
authors.affiliation: Novartis Pharmaceuticals Corporation, Florham Park, NJ, USA.
authors.forename: Jinhee
authors.initials: J
authors.lastname: Park
authors.affiliation: Novartis Pharma AG, Basel, Switzerland.
authors.forename: Fabian
authors.initials: F
authors.lastname: Herbst
authors.affiliation: Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
authors.forename: Jennifer L
authors.initials: JL
authors.lastname: Beaumont
authors.affiliation: University Hospital Leuven, Leuven Cancer Institute, Leuven, Belgium.
authors.forename: Oliver
authors.initials: O
authors.lastname: Bechter
mesh_terms: D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000494:pharmacology / Q000627:therapeutic use*; D000068338:Everolimus / Q000008:administration & dosage / Q000494:pharmacology / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D008297:Male; D011788:Quality of Life
chemical_list: D000970:Antineoplastic Agents; D000068338:Everolimus