Efficacy and safety of targeted agents, such as everolimus and sunitinib, have been demonstrated in the prospective trials for patients with GEP-NET. Considering heterogeneous clinical features of NET, evaluation of real-world outcomes with these agents are necessary. We retrospectively analyze the treatment outcomes of everolimus and sunitinib for patients (pts) with GEP-NET.
Between Mar 2007 and Oct 2014, a total of 44 GEP-NET pts treated with everolimus or sunitinib were included. Considering distinct characteristics between pancreatic (Pan) and non-PanNETs, efficacy analysis was performed separately, while safety analysis included all pts.
PanNET was most common type (n = 28, 64%) and followed by hindgut NET (n = 11, 25%) and foregut NET (n = 5, 11%). Sunitinib and everolimus were given in 27 (61%) and 17 (39%) pts, respectively. Among 41 pts that pathology review was available, tumor grade (G) was G1/2 in 36 (78%) and G3 in 5 (12%). Cytotoxic chemotherapy and somatostatin analogue were previously given in 16 (36%) and 18(41%) pts, respectively. In pts with PanNET, median progression-free survival (PFS) with everolimus and sunitinib was 16.6 months (95% CI, 8.0-25.1) and 8.0 months (95% CI, 0.0-17.4), and there was no significant difference between two agents (p = 0.51). For non-PanNET pts, median PFS was 14.7 months (95% CI, 2.4-27.0) with everolimus and 1.7 months (95% CI, 0.5-3.0; p = 0.001) with sunitinib; G3 tumor and prior cytotoxic chemotherapy were more common in pts with sunitinib than those with everolimus (30% vs 0%, and 70% vs 50%, respectively).Treatment was discontinued due to the adverse events in 3 pts (14%) with sunitinib and 4 (29%) with everolimus. Most common grade 3-4 toxicities were neutropenia (n = 9, 33%), anemia (5, 19%), diarrhea (3, 11%), and hand-foot syndrome (2, 7%) in pts with sunitinib (n = 27), and pneumonitis (2, 12%), and thrombocytopenia/stomatitis (1, 6%) in those with everolimus (n = 17). Tumor grade was a significant predictive factor for PFS (G1/2: median 14.7 months vs G3: 2.5 months, p = 0.002).
Both everolimus and sunitinib were well tolerable and effective in GEP-NET pts. The activity of everolimus was seen across all GEP-NETs and consistent with previous trials.
Clinical trial identification
Legal entity responsible for the study
Asan Medical Center (AMC) IRB
All authors have declared no conflicts of interest.