TABLE 1.
Main randomized clinical trials of ICIs for advanced HCC.
| Name | Treatment | Study phase | Control group | Primary endpoint | ORR, % | Median OS, months | Median PFS, months |
|---|---|---|---|---|---|---|---|
| CheckMate 040 | Nivolumab | I/II | None | Safety and ORR | 20 | 15.6 | 4.0 |
| CheckMate 040 | Nivolumab plus ipilimumab | I/II | None | Safety, tolerability, and ORR | 32 | 22.8 | NR |
| KEYNOTE-224 | Pembrolizumab | II | None | ORR | 17 | 12.9 | 4.9 |
| KEYNOTE-240 | Pembrolizumab | III | Placebo | OS and PFS | 18.3 vs. 14.4, p < 0.001 | 13.8 vs. 10.6, p = 0.024 | 3.0 vs. 2.8, p = 0.002 |
| CheckMate 459 | Nivolumab | III | Sorafenib | OS | 15 vs. 7, p = NR | 16.4 vs. 14.8, p = 0.052 | 3.7 vs. 3.8, p = NS |
| IMbrave150 | Atezolizumab plus bevacizumab | III | Sorafenib | OS and PFS | 30 vs. 11, p < 0.001 | 19.2 vs. 13.4, p < 0.001 | 6.8 vs. 4.3, p < 0.001 |
| COSMIC-312 | Cabozantinib plus atezolizumab | III | Sorafenib | OS and PFS | 13 vs. 6, p = NR | 15.4 vs. 15.5, p = 0.44 | 6.8 vs. 4.2, p = 0.001 |
ICI, immune checkpoint inhibitor; NR, not reported; NS, not significant; ORR, objective response rate; OS, overall survival; PFS, progression-free survival.