Table 1. Published studies on FDA-approved immune checkpoint inhibitors in HCC and BTC.

Study name	Year	Medication	Disease	Setting	Study design/study size	Outcomes
KEYNOTE-016 (14)	2017	Pembrolizumab	dMMR deficient solid tumors	Unresectable or metastatic, later-line	Phase II, 86	ORR 53%, CR 21%; DCR 77%; mPFS and mOS NR
CheckMate-040 (12)	2017	Nivolumab	HCC	Advanced, second-line‡	Phase I/II, 262	ORR 20%, CR 1%; DCR 64%; mDOR 9.9 mos; mPFS 4.0 mos; grade 3–5 TRAEs 19%
KEYNOTE-224 (15)	2018	Pembrolizumab	HCC	Advanced, second-line†	Phase II, 104	ORR 17%, CR 1%; DCR 69%; mDOR 9 mos; mPFS 7.0 mos; grade 3–5 TRAEs 26%

^†, Child Pugh A only; ^‡, Child Pugh A or B7. HCC, hepatocellular carcinoma; BTC, biliary tract cancer; dMMR, mismatch repair deficient; ORR, objective response rate; CR, complete response; mPFS, median progression-free survival; mOS, median overall survival; DCR, disease control rate; mDOR, median duration of response; mos, months; TRAEs, treatment-related adverse effects; NR, not reported.