Table 2.
Reported outcomes of single-agent immune checkpoint inhibitors in advanced biliary tract cancer (BTC).
| Phase | Setting, (Type of BTC) | ICI | Agents Description | Number of Patients | Outcomes |
|---|---|---|---|---|---|
| Ib [36] | Second- or later-line (iCCA, eCCA, GBC) | Pembrolizumab | Pembrolizumab: PD-1 inhibitor | 24 (all patients had PD-L1 ≥1%) | mPFS 1.8 months |
| mOS 5.7 months | |||||
| ORR 13% | |||||
| SD rate 17% | |||||
| II [36] | Second- or later-line (iCCA, eCCA, GBC) | Pembrolizumab | Pembrolizumab: PD-1 inhibitor | 104 (61 patients had PD-L1 ≥1%) | mPFS 2.0 months |
| mOS 7.4 months | |||||
| ORR 5.8% (6.6% in PD-L1+; 2.9% in PD-L1-) | |||||
| II [40] | Second- or later-line (iCCA, eCCA, GBC) | Nivolumab | Nivolumab: PD-1 inhibitor | 30 | mPFS 1.4 months |
| mOS 5.2 months | |||||
| PR rate 3% | |||||
| II [37] | Second- or later-line (iCCA, eCCA, GBC) | Nivolumab | Nivolumab: PD-1 inhibitor | 54 | mPFS 3.7 months |
| mOS 14.2 months | |||||
| ORR 22% | |||||
| DCR 50% | |||||
| II [41] | Second- or later-line (iCCA, eCCA, GBC) | Durvalumab | Durvalumab: PD-L1 inhibitor | 42 | mPFS 1.5 months |
| mOS 8.1 months | |||||
| PR rate 4.8% | |||||
| I [42] | Second- or later-line (iCCA, eCCA, GBC) | M7824 | M7824: PD-L1 inhibitor | 30 | mOS 12.7 months |
| ORR 20% |
AVC, ampulla of Vater cancer; DCR, disease control rate; eCCA, extrahepatic cholangiocarcinoma; GBC, gallbladder cancer; iCCA, intrahepatic cholangiocarcinoma; m, median; ORR, overall response rate; OS, overall survival; PD-1, programmed death 1; PFS, progression-free survival; PR, partial response; SD, stable disease.