From: Emerging therapies in advanced hepatocellular carcinoma
Trial | Phase | Mechanism of action | Primary endpoint | OS (months) | PFS (months) | ORR (%) |
---|---|---|---|---|---|---|
Regorafenib vs placebo (RESORCE) | III | Multi-targeted TKI | OS | 10.6 vs 7.8 months (p < 0.0001) | 3.1 vs 1.5 months (p < 0.0001) | 11 vs 4 (p = 0.0047) |
Cabozantinib vs placebo (CELESTIAL) | III | Multi-targeted TKI | OS | 10.2 vs 8.0 months (p = 0.0049) | 5.2 vs 1.9 months (p < 0.001) | 4 vs 0.4 (p = 0.0086) |
Nivolumab | I/II | PD-1 immune checkpoint inhibitor | ORR | 15 months | – | 20 (95% CI = 15–26) |
Pembrolizumab | II | PD-1 immune checkpoint inhibitor | ORR | – | 4.8 months | 16.3 |
Ramucirumab vs placebo | III | Anti-VEGF | OS | 9.2 vs 7.6 months (p = 0.14) | 2.8 vs 2.1 months (p < 0.0001) | 7 vs < 1 (p < 0.0001) |
Axitinib + BSC vs placebo + BSC | II | Anti-VEGF | Tumor control | 12. 7 vs 9.7 months (p = 0.287) | 3.6 vs 1.9 months (p = 0.004) | 9.7 vs 2.9 (p = 0.091) |
Brivanib vs placebo | III | FGFR inhibitor | OS | 9.4 vs 8.2 months (p = 0.3307) | 4.2 vs 2.7 months (p < 0.001) | 10 vs 2 |
Everolimus vs placebo | III | mTOR inhibitor | OS | 7.6 vs 7.3 months (p = 0.68) | 3 vs 2.6 months | 8 vs 3 |