Immunotherapy in biliary tract cancers: Current evidence and future perspectives

Table 1 Immunotherapy studies including biliary cancer patients, biomarkers, response rate and Food and Drug Administration approvals

Ref.	Patients	n	Biomarker	RR	Drug	FDA approval
Lemery et al[26], 2017	Previously treated	11	MSI-H	27%	Pembrolizumab	5/23/2017
Marabelle et al[27], 2020 (KN158)	Previously treated	22	MSI-H	40.9%	Pembrolizumab	5/23/2017
Marabelle et al[31], 2020 (KN158)	No TMB-H in cholangiocarcinoma cohort	0	TMB-H (≥ 10 mut/Mb)	?	Pembrolizumab	6/16/2020
Ueno et al[32], 2018 (KN158)	Previously treated	104	PD-L1 (CPS ≥ 1)	CPS ≥ 1: 6.6%CPS < 1: 2.9%	Pembrolizumab	-
Bang et al[33], 2019 (KN028)	Previously treated	24	PD-L1 ≥ 1	13%	Pembrolizumab	-
Kim et al[35], 2020	Previously treated	54	-	IA: 22%CIR: 11%	Nivolumab	-
Klein et al[40], 2020 (CA 209-538)	Previously treated	39	-	24%	Nivolumab + Ipilimumab	-
Oh et al[42], 2020	Chemo-naïve	121	-	50%-73%	Gem + Cis + Durvalumab ± Tremelimumab	-
Oh et al[19], 2022 (TOPAZ-1)	Chemo-naive	344	PD-L1 (TAP)	18.7%	Gem + Cis	-
		341		26.7%	Gem + Cis + Durvalumab

KN: Keynote; RR: Response rate; MSI-H: Microsatellite instability-high; TMB-H: Tumor mutational burden-high; mut/Mb: Mutations/megabase; PD-L1: Programmed death-ligand 1; CPS: Combined positive score; IA: Investigator-assessed; CIR: Central independent review; Gem: Gemcitabine; Cis: Cisplatin; TAP: Tumor area positivity.