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Table 2  Selected clinical trial for advanced HCC in the second line

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
  1. TKI tyrosine kinase inhibitor, PD-1 programmed cell death protein-1, VEGF vascular endothelial growth factor, FGFR fibroblast growth factor receptor, mTOR mechanistic target of rapamycin, OS overall survival, ORR objective response rate, PFS progression free survival