Table 1.
Summary of published data regarding mTOR inhibitors in advanced BTC.
| Compound(s) | Phase | Patients | Response | Survival |
|---|---|---|---|---|
| Everolimus, 1 L [106] | Case report | iCCA (n = 1) with PIK3CA mutation | PR | PFS > 6 m |
| Everolimus [107] | Phase I | Advanced BTC (n = 22) | DCR: 50% (11/22) | NA |
| Everolimus (>2 L) [108] | Phase II | Advanced BTC (n = 39) | DCR: 44.7% RR: 5.1% (including 1 CR) |
mPFS: 3.2 m (1.8–4.0) mOS: 7.7 m (5.5–13.2) |
| Everolimus [109] | Phase II | CCA (n = 1), PTEN loss | SD | NA |
| Everolimus (1 L) [110] | Phase II | Advanced BTC (n = 27) | DCR at 12 weeks: 48% PR: 12% (3/25) SD: 60% (15/25) |
mPFS: 5.5 m (2.2–10.0) mOS: 9.5 m (5.5–16.6) |
| Sirolimus [111] | Phase II | iCCA (n = 9) | SD: 33% (3/9) PD: 67% (6/9) |
mOS:7 (2.6–35) |
| Sirolimus [112] | Phase II | hilar CCA (n = 1) with PIK3CA mutation | PD | PFS: 0.9 m |
| Everolimus, gemcitabine, cisplatin (1 L) [113] | Phase I | Cohort III, CCA and GBC (n = 10) | SD: 60% (6/10) PD: 40% (4/10) |
NA |
1 L, first line; 2 L, second line; iCCA, intrahepatic cholangiocarcinoma; GBC, gallbladder cancer; BTC, biliary tract cancer; CR, complete response; PR, partial response; SD, stable disease; PD, progressive disease; DCR, disease control rate; mPFS, median progression-free survival; mOS, median overall survival.