Outcomes of patients treated by FGFR inhibitors according to alteration types: An agnostic monocentric retrospective cohort.

Authors

Cedric Pobel, Hassan Badreddine, Cristina Smolenschi, Alice Boileve, Michel Ducreux, Luc Friboulet, Rastislav Bahledal, Anas Gazzah, Kristi Beshiri, Marie Porte, Imen Ben-Ammar, Marie-Anne Audisio, Kaissa Ouali, Mohamed Bani, Yohann Loriot, Damien Vasseur, Maximilliano Gelli, Christophe Massard, Francesco Facchinetti, Antoine Hollebecque

Published in

European journal of cancer (Oxford, England : 1990). Volume 244. Pages 116900. Jun 19, 2026. Epub Jun 19, 2026.

Abstract

Fibroblast growth factor receptor (FGFR) alterations including mutations, fusions and amplifications, are known oncogenic drivers. FGFR tyrosine kinase inhibitors (FGFRi) have demonstrated clinical activity in patients with FGFR3-driven urothelial carcinoma and FGFR2-driven cholangiocarcinoma. This study aims to describe outcomes with FGFRi according to alteration and tumor type.
We retrospectively assessed data from patients with FGFR alterations treated by an FGFRi in multiple clinical trials and in routine care at Gustave Roussy.
Among 158 patients treated by an FGFRi from February 2011 to March 2025, 27 had FGFR amplifications, 76 FGFR fusions, and 55 FGFR mutations. FGFR fusions, most common in cholangiocarcinoma, were associated with the most favorable outcomes with an overall response rate (ORR) of 45.9%, median progression free survival (PFS) and overall survival (OS) of 7.3 and 19.7 months, respectively. FGFR amplifications, found predominantly in breast cancer, were associated with the poorest outcomes (ORR of 11.1%, median PFS and OS of 2.1 and 9.9 months, respectively). FGFR mutations, observed mainly in urothelial carcinoma, showed intermediate efficacy (ORR of 30.9%, median PFS and OS of 4.7 and 12.4 months, respectively). Among the 22 patients who received a second FGFRi, outcomes were inferior to firsts exposure (ORR of 13.6% vs 47.6% and median PFS of 3.8 vs 8.5 months).
Clinical activity of FGFRi was heterogenous across FGFR alteration and tumor types. FGFR fusions and mutations were associated with greater benefit, whereas amplifications were not. Further investigation of resistance mechanisms and development of next-generation FGFRi are needed.

PMID:
42330567
Bibliographic data and abstract were imported from PubMed on 23 Jun 2026.

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