PD-17 Multicentric prospective study assessing clinical benefit of targeted treatment for patients with biliary tract cancers

Biliary Tract Cancers (BTC) patients (pts) have poor prognosis and limited therapeutic options, but there is increasing evidence for biomarker-directed therapy, with several potentially actionable molecular targets FDA-approved drugs. ESMO ASCO recommends the use of tumour multigene next generation sequencing (NGS) BTC. This was a multicentric study assessing real-life clinical actionability alterations identified comprehensive NGS affected by advanced BTCs treated at five Italian referral centers. performed either via “Hotspot Cancer Panel, Ion Torrent®”, “Oncomine Comprehensive Assay Plus” or FoundationOne®CDx panel; FGFR2 testing also fluorescence in situ hybridisation Archer®FusionPlex®Oncology Research Panel. Molecular were correlated targeted treatments administered efficacy endpoints. Benefit from treatment evaluated means progression-free survival (PFS) ratio, as defined ratio each patient’s PFS2 (i.e. PFS on molecularly informed therapy) to PFS1 (PFS his/her most recent previous treatment), delta equal 1. Out 587 pts adequate tissue NGS, 397 (68%) intrahepatic (iCCA), 58 (10%) perihilar (pCCA) 63 (11%) distal (dCCA) cholangiocarcinoma, 64 gallbladder 5 (1%) ampullary cancers. Targeted therapy actually started 95 (16%), 15 (16%) ivosidenib IDH1 mutations, (66%) inhibitors alterations, 3 (3%) immune checkpoint (ICIs) MSI-H, 9 BRAF+MEK BRAFV600E mutations 4 (4%) HER2 amplification. Overall response rate disease control 20% 61%, respectively. Median 5.5 months (95% CI 4.1-7.3) overall 3.0 ivosidenib, 5.8 inhibitors, 9.5 ICIs 7.3 inhibitors. PFSratio 1.02 (95%CI 0.75-1.34), benefit 52%. Of note, 34 (36%) received ≥ III line. start 11.4 9.4-15.2) months. feasible daily practice it should be performed, recommended, all BTC, since has clinically meaningful pretreated patients.