Authors
Carolina Liguori, Riccardo Giampieri, Blandine Delaunay, Giada Pinterpe, Adelaide Mazzocca, Antoine Hollebecque, Jean Frederic Blanc, Mohamed Bouattour, Eric Assenat, Meher Ben Abdelghani, Matthieu Sarabi, Monica Niger, Caterina Vivaldi, Mario Mandalà, Andrea Palloni, Maria Bensi, Silvio Ken Garattini, David Tougeron, Pierre Combe, Massimiliano Salati, Margherita Rimini, Andrea Casadei-Gardini, Chiara Alessandra Cella, Marco Tucci, Anna Diana, Elena Mori, Raffaella Longarini, Pascal Artru, Gael Roth, Ludovic Evesque, Agathe Vienne, Anthony Turpin, Sandrine Hiret, Vincent Bourgeois, Camille Herve, Rodolphe Paulon, Marion Stacoffe, David Malka, Matthieu Delaye, Julien Edeline, Astrid Lievre, Rosine Guimbaud, Rita Balsano, Nadim Fares, Rossana Berardi, Alessandro Parisi
Published in
Liver international : official journal of the International Association for the Study of the Liver. Volume 46. Issue 8. Pages e70813.
Abstract
Anti-FGFR therapies changed the treatment landscape for patients with previously treated, advanced, or metastatic cholangiocarcinoma (CCA) harbouring FGFR2 fusions/rearrangements. However, primary resistance to FGFR inhibitors remains a key challenge. In the present real-world study, we aimed to evaluate the prognostic impact of concomitant GAs in patients with FGFR2-positive CCA treated with pemigatinib.
This study included PEMIREAL-PEMIBIL patients treated with pemigatinib in second or later lines. Only patients who underwent extensive DNA- and/or RNA-based next-generation sequencing (NGS) analysis were considered. Primary endpoint was progression-free survival (PFS), with overall response rate, disease control rate and overall survival (OS) as secondary endpoint. OS and PFS were calculated by Kaplan-Meier and log-rank test. Multivariate analysis used cox-regression model. Level of statistical significance p was 0.05.
Of 72 patients of PEMIREAL-PEMIBIL, 63 patients had evaluable NGS data, with concomitant GAs identified in 28 patients (44.4%). The most frequently observed concomitant GAs involved BAP1 (7/63, 11.1%), CDKN2A (7/63, 11.1%), TP53 (6/63, 9.5%), CDKN2B (5/63, 7.9%), PTEN (3/63, 4.7%) and IDH1 (1/63, 1.5%). A significantly shorter PFS was observed in patients with CDKN2A mutations compared to CDKN2A wild-type tumours (4.79 vs. 8.66 months, p = 0.0011, HR: 3.48 95% CI: 0.91-13.24), and similarly in patients with BAP1 mutations compared to BAP1 wild-type tumours (5.97 vs. 8.52 months, p = 0.025, HR:2.55 95% CI: 0.72-9.00). No significant differences in OS were observed.
Our results support the negative prognostic role of BAP1 and CDKN2A GAs on PFS in patients with locally advanced or metastatic CCA with FGFR2 gene fusion/rearrangement treated with pemigatinib in a real-world setting.
PMID:
42494243
Bibliographic data and abstract were imported from PubMed on 24 Jul 2026.
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