Table 3.

Summary of main clinical trials evaluating immune checkpoint inhibitors in biliary tract cancer patients with advanced disease.

Treatment Arm	Agents Description	NCT Name	Phase	Setting	Results
Pembrolizumab	Pembrolizumab: PD-1 inhibitor	NCT02628067 (KEYNOTE-158) [93]	II	Second- or later-line	ORR: 5.8% (2.1–12.1) mOS: 7.4 mo (5.5–9.6) mPFS: 2.0 mo (1.9–2.1)
Pembrolizumab	Pembrolizumab: PD-1 inhibitor	NCT02054806 (KEYNOTE-028) [93]	Ib	Second- or later-line	ORR: 13.0% (2.8–33.6) mOS: 5.7 mo (3.1–9.8) mPFS: 1.8 mo (1.4–3.7)
Nivolumab	Nivolumab: PD-1 inhibitor	JapicC- TI-153098 [96]	I	Second- or later-line	ORR: 3.3% (0.7–13.6) mOS: 5.2 mo (4.5–8.7) mPFS: 1.4 mo (1.4–1.4)
Nivolumab	Nivolumab: PD-1 inhibitor	NCT02829918 [94]	II	Second- or later-line	PR: 22% DCR: 59% mOS: 14.2 mo (5.98-not reached) mPFS: 3.7 mo (2.3–5.69)
Durvalumab	Durvalumab: PD-L1 inhibitor	NCT01938612 [99]	I	First- or later-line	DCR: 16.7% mOS: 8.1 mo (5.6–10.1)
Nivolumab plus ipilimumab	Nivolumab: PD-1 inhibitor Ipilimumab: CTLA-4 inhibitor	NCT02923934 (CA209–538) [100]	II	First- or later-line	ORR: 23% DCR: 44% mOS: 5.7 mo (2.7–11.9) mPFS: 2.9 mo (2.2–4.6)
Durvalumab plus tremelimumab	Durvalumab: PD-L1 inhibitor Tremelimumab: CTLA-4 inhibitor	NCT01938612 [99]	I	First- or later-line	DCR: 32.2% mOS: 10.1 mo (6.2–11.4)
Nivolumab plus CisGem	Nivolumab: PD-1 inhibitor	NCT03311789 [95]	II	First-line	ORR: 55.6% DCR: 92.6% mOS: 8.5 mo (5.0–12.5) mPFS: 6.1 mo (3.4–8.2)
Nivolumab plus CisGem	Nivolumab: PD-1 inhibitor	JapicCTI- 153098 [96]	I	First-line	ORR: 36.7% mOS: 15.4 mo (11.8-NE) mPFS: 4.2 mo (2.8–5.6)

CisGem, cisplatin plus gemcitabine combination; CTLA-4, Cytotoxic T-Lymphocyte Antigen 4; DCR: disease control rate; NE: not estimable; ORR, overall response rate; mo: months; mOS, median overall survival; PD-1, programmed death 1, mPFS: median progression-free survival; PR: partial response.