Table 2.
Efficacy and safety data of immune checkpoint inhibitors in advanced hepatocellular carcinoma.
| Agent (mechanism) | Trial | Phase | Design | n | Target population | Response rate |
Median survival |
Grade 3/4 AEs | ||
|---|---|---|---|---|---|---|---|---|---|---|
| ORR | DCR | OS | PFS | |||||||
| Monotherapy | ||||||||||
| Nivolumab (anti-PD1) |
CHECKMATE 040138 (NCT 01658878) |
I/II | Cohort 1 (dose escalation)/Cohort 2 (dose expansion) | 48/214 | Advanced HCC: HCV, HBV or non-infected; sorafenib-naïve or treated |
15% /20% |
58% /64% |
15 /NR |
NR /4 |
25%/19% |
| Pembrolizumab (anti-PD1) |
KEYNOTE 224140 (NCT02702414) |
II | Non-randomised, single-arm | 104 | Advanced HCC: sorafenib-treated |
17% | 62% | 12.9 | 4.9 | 25% |
| Tremelimumab (anti-CTLA4) |
NCT01008358151 | II | Non-randomised, single-arm | 21 | Inoperable HCC: Naïve or previously treated |
17.6% | 76.4% | 8.2 | NR | 45% |
| Durvalumab (anti-PDL1) |
NCT01693562⁎,152 | II | Non-randomised, single arm | 40 | Stage III or IV Fail, ineligible, refusal or progression to first-line |
10.3 | NR | 13.2 | 2.7 | 20% |
| Combination | ||||||||||
| Atezolizumab (anti-PDL1)+ Bevacizumab (anti-VEGF) |
NCT02715531⁎,144 | Ib | Non-randomised, single-arm | 103 | Unresectable HCC: Non-previous treated; HBV, HCV or non-infected |
32% | 96% | NR | 14.9 | 28% |
| Lenvatinib (kinase inhibitor)+ Pembrolizumab (anti-PD1) |
NCT03006926⁎,143 | Ib | Non-randomised, single arm | 30 | Unresectable HCC: sorafenib-naïve or treated; HCV, HBV or non-infected. |
46% 26.9 | 92% | NR | 9.69 | 60% |
AEs, adverse events; DCR, disease control rate; HCC, hepatocellular carcinoma; HBV, hepatitis B virus; HCV, hepatitis C virus; NR, not reported; ORR, overall response rate; PFS, progression-free survival; PD1, programmed cell-death.
Median survival given in months
*
non-published data.