Relevant targetable aberrations in BTCs
| Molecular alteration | Incidence (%) | Anatomical location | Investigated agents |
|---|---|---|---|
| FGFR2 gene fusions | 14–23 | iCCA | PemigatinibInfigratinibFutibatinibDerazantinibDebio 1347Erdafitinib |
| IDH mutations | 7–20 (IDH1)3 (IDH2) | iCCA | IvosidenibEnasidenibDasatinibFT-2102 |
| HER2 overexpression/amplification | 15 | GBC, eCCA | TrastuzumabTrastuzumab-pertuzumabTrastuzumab deruxtecanT-DM1Zanidatamab |
| HER2 mutations | 1–2 | GBC, eCCA, iCCA | Neratinib |
| 7 | AVC | ||
| BRAF V600E mutation | < 5 | iCCA | Dabrafenib-trametinib |
| NTRK fusions | 3.5 | iCCA | LarotrectinibEntrectinib |
| MSI-H | 1 | eCCA, iCCA, GBC | Pembrolizumab |
T-DM1: trastuzumab-emtansine; FT-2102: olutasidenib