Table 1.
Clinical trials of targeted therapies in biliary tract cancer
| Agent | Pathway | Trial Phase | ORR | PFS | OS |
|---|---|---|---|---|---|
| Erlotinib [37] | EGFR (TKI) | II (single-arm) | 8% | 6-month PFS: 17% | 7.5 months |
| Gemcitabine + oxaliplatin +/− continuous erlotinib [38] | EGFR (TKI) | III (randomized) | 30% vs. 16% | 5.8 vs. 4.2 months | 9.5 vs. 9.5 months |
| Gemcitabine + oxaliplatin + pulsed erlotinib [39] | EGFR (TKI) | Ib (single-arm) | 24% | 6-month PFS: 75% | NR |
| Gemcitabine + oxaliplatin + cetuximab [40] | EGFR (mAb) | II (single-arm) | 63% | 8.8 months | 15.2 months |
| Gemcitabine + oxaliplatin +/− cetuximab [41] | EGFR (mAb) | II (randomized) | 23 vs. 29% | 6.0 vs. 5.3 months | 11.0 vs. 12.4 months |
| Gemcitabine + oxaliplatin + capecitabine + panitumumab [42] | EGFR (mAb) | II (single-arm) | 33% | 8.3 months | 9.8 months |
| Gemcitabine + oxaliplatin + bevacizumab [43] | VEGF (mAb) | II (single-arm) | 40% | 7.0 months | 12.7 months |
| Erlotinib + bevacizumab [44] | EGFR (TKI) + VEGF (mAb) | II (single-arm) | 18.4% | TTP: 4.4 months | 9.9 months |
| Sorafenib [45] | VEGF (TKI) | II (single-arm) | 2% | 2.3 months | 4.4 months |
| Sorafenib +/− gemcitabine [46] | VEGF (TKI) | II (randomized) | 2.7% vs. 0% | 2.9 vs. 2.3 months | 6.5 vs. 4.3 months |
| Gemcitabine +/− sorafenib [47] | VEGF (TKI) | II (randomized) | 7% | 2.9 months | 9.4 months |
| Sunitinib [48] | VEGF (TKI) | II (single-arm) | 8.9% | TTP: 1.7 months | 4.8 months |
| Lapatinib [50] | HER2 (TKI) | II (single-arm) | 0% | 1.8 months | 5.2 months |
| Selumetinib [51] | MEK (TKI) | II (single-arm) | 12% | 3.7 months | 9.8 months |
ORR overall response rate, PFS progression-free survival, OS overall survival, EGFR epidermal growth factor receptor, VEGF vascular endothelial growth factor, HER2 human epidermal growth factor receptor 2, MEK mitogen-activated protein kinase/extracellular-signal regulated kinase, TKI tyrosine kinase inhibitor, mAb monoclonal antibody, TTP time to progression, NR not reported