Table 1.
Principal characteristics of phase II studies.
| Study | Phase | Design | ICI | Target | Sample Size * | ORR No. (%) |
Median PFS, mos (Range) | Median OS, mos (Range) |
|---|---|---|---|---|---|---|---|---|
| Mehnert [26] (NCT02054806) |
1b | Multicohort: epNET | Pembrolizumab | PD-1 | 25 | 3 (12) | 5.6 (3.5–10.7) | 21.1 (9.1–22.4) |
| Mehnert [26] (NCT02054806) |
1b | Multicohort: pNET | Pembrolizumab | PD-1 | 16 | 1 (6) | 4.5 (3.6–8.3) | 21.0 |
| Strosberg [27] (NCT02628067) |
2 | Single cohort: mixNET | Pembrolizumab | PD-1 | 107 | 4 (4) | 4.1 (3.5–5.4) | 24.2 (15.8–32.5) |
| Yao [28] (NCT02955069) |
2 | Multicohort: epNET | Spartalizumab | PD-1 | 62 | 6 (10) | - | - |
| Yao [28] (NCT02955069) |
2 | Multicohort: pNET | Spartalizumab | PD-1 | 33 | 1 (3) | - | - |
| Yao [28] (NCT02955069) |
2 | Multicohort: mixNEC | Spartalizumab | PD-1 | 21 | 1 (5) | - | - |
| Patel [29] (NCT02834013) |
2 | Single cohort: epNEN | Nivolumab + ipilimumab | PD-1, CTLA-4 | 32 | 8 (25) | 4.0 (3.0–6.0) | 11 |
| Lu [30] (NCT03167853) |
1b | Multicohort: mixNEC, mixNET/pNEN, epNEN, mixNEN | Toripalimab | PD-1 | 40 | 8 (20) | 2.5 (1.9–3.1) | 7.8 (5.0–10.8) |
| Vijayvergia [31] (NCT02939651) |
2 | Single cohort: mixNEN | Pembrolizumab | PD-1 | 29 | 1 (3) | 2.0 (1.5–2.4) | 4.7 |
| Halperin [32] (NCT03074513) |
2 | Multicohort: pNET | Atezolizumab + bevacizumab | PD-L1, TKI | 20 | 4 (20) | 19.6 | - |
| Halperin [32] (NCT03074513) |
2 | Multicohort: epNET | Atezolizumab + bevacizumab | PD-L1, anti-VEGF | 20 | 3 (15) | 14.9 | - |
| Zhang [33] (NCT03167853) |
1b | Multicohort: mixNEC, mixNET | Toripalimab | PD-1 | 21 | 6 (29) | 2.8 (1.6–4.0) | - |
| Fottner [34] (NCT03352934) |
2 | Single cohort: mixNEN | Avelumab | PD-L1 | 29 | 2 (7) | - | 4.2 (1.0–12.0) |
| Mulvey [35] (NCT03136055) |
2 | Single cohort: epNEC | Pembrolizumab | PD-1 | 13 | 1, 8 | 2.0 | - |
| Frumovitz [6] (NCT02721732) |
2 | Single cohort: epNEC | Pembrolizumab | PD-1 | 7 | 0 (0) | 2.1 (0.8–3.3) | - |
| Rodriguez-Freixinos [37] (NCT03278405) |
2a | Single cohort: epNEC | Avelumab | PD-L1 | 9 | 0 (0) | 3.0 (1.0–10.0) | 5.0 (2.0–15.0) |
| Klein [38] (NCT02923934) |
2 | Single cohort: mixNEN | Nivolumab + ipilimumab | PD-1, CTLA-4 | 29 | 7 (24) | 4.8 (2.7–10.5) | 14.8 (4.1–21.2) |
| Capdevila [39] (NCT03095274) |
2 | Multicohort:epNET | Durvalumab + tremelimumab | PD-L1, CTLA-4 | 27 | 0 (0) | 5.3 (4.5–6.0) | - |
| Capdevila [39] (NCT03095274) |
2 | Multicohort:epNET | Durvalumab + tremelimumab | PD-L1, CTLA-4 | 31 | 0 (0) | 8 (4.9–11.1) | - |
| Capdevila [39] (NCT03095274) |
2 | Multicohort:pNET | Durvalumab + trremelimumab | PD-L1, CTLA-4 | 32 | 2 (6) | 8.1 (3.8–12.4) | - |
| Capdevila [39] (NCT03095274) |
2 | Multicohort:mixNEN | Durvalumab + tremelimumab | PD-L1, CTLA-4 | 33 | 2 (6) | 2.5 (2.1–2.7) | - |
* The sample size refers solely to patients evaluable for response. ICI: immune checkpoint inhibitor; ORR: objective response rate; PFS: progression-free survival; OS: overall survival; mos: months; ep: extra-pancreatic; p: pancreatic; mix: pancreatic and extra-pancreatic.