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Table 3 The key reported clinical trials of of PD-1/PD-L inhibitors in patients with hepatocellular carcinoma and biliary tract cancer

From: PD-1/PD-L blockade in gastrointestinal cancers: lessons learned and the road toward precision immunotherapy

Tumor type Target Drug Phase and identification Sample size Clinical end point TRAEs Reference
HCC PD-L1 Durvalumab Phase I/II
NCT01693562
39 ORR 10.3%; DCR 33.3%; median OS 13.2 months; 9-month OS rate 62.3%; 12-month OS rate 56.4% All grades 80.0%, including fatigue, pruritus, elevated AST; Grades 3–4 20.0%, including elevated AST and elevated ALT. ASCO 2017 [131]
HCC PD-1 Nivolumab Phase I/II
NCT01658878
262 ORR 23% (sorafenib-naive), 16–19% (sorafenib-experienced); DCR 63% (sorafenib-naive); 12- month OS rate 73% (sorafenib-naive), 60% (sorafenib-experienced) All grade 77%; Grade ≥3 23.5%, including elevated AST and elevated ALT. ASCO 2017 [157]
HCC PD-1 Nivolumab Phase I/II
NCT01658878
262 ORR 20% (dose expansion phase), 23% (sorafenib-naive), 21% sorafenib-treated); median DOR: 9.9 months (dose expansion phase), DCR 64% (dose expansion phase); 9-month OS rate 74% (dose expansion phase) Grades 3–4 20% ASCO 2017 [158]
HCC PD-1 Nivolumab Phase I/II
NCT01658878
48 ORR 15%; median OS 15.1 months; median DOR 23.7 months; 12-month OS rate 59%; 18-month OS rate 48% All grade 77%, including rash and AST increase; Grades 3–4 20%, including AST increase, lipase and ALT increase ASCO 2016 [159]
HCC PD-1 Nivolumab Phase I/II
NCT01658878
39 ORR 23%; CR 5%; PR 18%; 6-month OS rate 72% Any grade 71%, including AST increase, amylase increase, rash, ALT and lipase increase; grades 3–4 17%, including AST increase, ALT increase and lipase increase ASCO 2015 [160]
HCC PD-L1 Durvalumab Phase I/II
NCT01693562
21 12-month DCR 21% Any grade (multiple cancer types) 33%, including fatigue, nausea, rash, vomiting, and pyrexia; grade ≥3 (multiple cancer types) 7% ASCO 2014 [61]
BTC PD-1 Pembrolizumab Phase Ib
NCT02054806
24 ORR 17%; SD 17%; PD 17% All grade 63%, including pyrexia and nausea; grades 3–4 17%, including anemia, autoimmune hemolytic anemia, colitis, and dermatitis ECCO 2015 [55]