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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