Safety and QOL in Patients with Advanced NET in a Phase 3b Expanded Access Study of Everolimus

Marianne Pavel*, Nicole Unger, Ivan Borbath, Sergio Ricci, Tsann Long Hwang, Thomas Brechenmacher, Jinhee Park, Fabian Herbst, Jennifer L. Beaumont, Oliver Bechter

*Corresponding author for this work

Research output: Contribution to journalJournal Article peer-review

30 Scopus citations

Abstract

Background/Aims: 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). Methods: 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. Results: 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. Conclusions: 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. Trial Registration: EudraCT no. 2010-023032-17 [Figure not available: see fulltext.]

Original languageEnglish
Pages (from-to)667-675
Number of pages9
JournalTargeted Oncology
Volume11
Issue number5
DOIs
StatePublished - 01 10 2016

Bibliographical note

Publisher Copyright:
© 2016, Springer International Publishing Switzerland.

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