TY - JOUR
T1 - Safety and QOL in Patients with Advanced NET in a Phase 3b Expanded Access Study of Everolimus
AU - Pavel, Marianne
AU - Unger, Nicole
AU - Borbath, Ivan
AU - Ricci, Sergio
AU - Hwang, Tsann Long
AU - Brechenmacher, Thomas
AU - Park, Jinhee
AU - Herbst, Fabian
AU - Beaumont, Jennifer L.
AU - Bechter, Oliver
N1 - Publisher Copyright:
© 2016, Springer International Publishing Switzerland.
PY - 2016/10/1
Y1 - 2016/10/1
N2 - 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.]
AB - 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.]
UR - http://www.scopus.com/inward/record.url?scp=84969760189&partnerID=8YFLogxK
U2 - 10.1007/s11523-016-0440-y
DO - 10.1007/s11523-016-0440-y
M3 - 文章
C2 - 27193465
AN - SCOPUS:84969760189
SN - 1776-2596
VL - 11
SP - 667
EP - 675
JO - Targeted Oncology
JF - Targeted Oncology
IS - 5
ER -