Response to: Cholangiocarcinoma patients with FGFR2 fusions/rearrangements but primary refractory to pemigatinib: the real challenge? DOI Creative Commons
Arndt Vogel, Hongtao Zhen,

M L Veronese

et al.

ESMO Open, Journal Year: 2024, Volume and Issue: 9(11), P. 103937 - 103937

Published: Oct. 17, 2024

Language: Английский

New systemic treatment paradigms in advanced biliary tract cancer and variations in patient access across Europe DOI Creative Commons
Lorenza Rimassa, Ángela Lamarca, Grainne M. O’Kane

et al.

The Lancet Regional Health - Europe, Journal Year: 2025, Volume and Issue: 50, P. 101170 - 101170

Published: Feb. 19, 2025

In recent years, treatment options for patients with advanced biliary tract cancer (BTC) have increased significantly due to the positive results from phase 2/3 clinical trials of immune checkpoint inhibitors, combined chemotherapy, and molecularly targeted agents. These advances led need molecular testing identify actionable alterations amenable therapies. However, these improvements brought them many questions challenges, including identification resistance mechanisms therapeutic sequences. this Series paper we aim provide an overview current systemic BTC, highlighting disparities in access innovative treatments across European countries, which lead inequalities possibilities treating BTC. We also discuss how ongoing collaborative projects, such as COST Action Precision-BTC-Network CA22125, supported by (European Cooperation Science Technology), linked Network Study Cholangiocarcinoma (ENSCCA), can help overcome improve scenario.

Language: Английский

Citations

2

ESMO Clinical Practice Guideline interim update on the management of biliary tract cancer DOI Creative Commons

A. Vogel,

Michel Ducreux

ESMO Open, Journal Year: 2024, Volume and Issue: 10(1), P. 104003 - 104003

Published: Dec. 17, 2024

Language: Английский

Citations

6

New systemic treatment options for advanced cholangiocarcinoma DOI Creative Commons
Valentina Zanuso, Giulia Tesini, Elena Valenzi

et al.

Journal of Liver Cancer, Journal Year: 2024, Volume and Issue: 24(2), P. 155 - 170

Published: Aug. 8, 2024

Cholangiocarcinoma (CCA) is a rare and aggressive cancer, mostly diagnosed at advanced or metastatic stage, which point systemic treatment represents the only therapeutic option. Chemotherapy has been backbone of CCA treatment. More recently, immunotherapy changed landscape, as immune checkpoint inhibitors have yielded first improvement in survival currently, addition either durvalumab pembrolizumab to standard care cisplatin plus gemcitabine new first-line However, use subsequent lines not demonstrated its efficacy therefore, it approved, except for selected microsatellite instability-high population. In addition, advances comprehensive genomic profiling led identification targetable genetic alterations, such isocitrate dehydrogenase 1 (IDH1), fibroblast growth factor receptor 2 (FGFR2), human epidermal (HER2), proto-oncogene B-Raf (BRAF), neurotrophic tropomyosin kinase (NTRK), rearranged during transfection (RET), Kirsten rat sarcoma virus (KRAS), mouse double minute homolog (MDM2), thus favoring development precision medicine approach previously treated patients. Despite these advances, molecularly driven agents limited subgroup This review aims provide an overview newly approved therapies, ongoing studies, future research challenges management.

Language: Английский

Citations

4

Current status and prospects of targeted therapy for cholangiocarcinoma based on molecular characteristics DOI
Xiaoqiang Cui, Teng Huang, Tianyi Jiang

et al.

Cancer Letters, Journal Year: 2025, Volume and Issue: 614, P. 217540 - 217540

Published: Feb. 7, 2025

Language: Английский

Citations

0

Clinical characterization and therapeutic targeting of fusion genes in oncology DOI Creative Commons
Susan Morand, Lauren Elizabeth Rager, Daniel J. Craig

et al.

Future Oncology, Journal Year: 2025, Volume and Issue: unknown, P. 1 - 12

Published: March 24, 2025

Gene fusions represent important oncogenic driver mutations resulting in aberrant cellular signaling. In up to 17% of all solid tumors at least one gene fusion can be identified. Precision therapy targeting signaling has demonstrated effective clinical benefit. Advancements clinically relevant next-generation sequencing and bioinformatic techniques have enabled expansion therapeutic opportunity subpopulations patients with expression. Clinically, tyrosine inhibitors shown efficacy treating expressing cancers. Fusion genes are also clonal mutations, meaning it is a personal cancer target involving cells that patient, not just subpopulation within the mass. Thus, both signal disruption immune directions. This review discusses targeting, resistance, molecular biomarkers.

Language: Английский

Citations

0

Efficacy and Toxicity of Pemigatinib in Advanced Cholangiocarcinoma Harboring FGFR Fusions or Rearrangements: A Systematic Review and Meta-analysis DOI
Erman Akkuş, Arzu Yaşar, Lorenza Rimassa

et al.

Targeted Oncology, Journal Year: 2025, Volume and Issue: unknown

Published: April 13, 2025

Language: Английский

Citations

0

Clinical Use of Comprehensive Genomic Profiling Tests in Intrahepatic Cholangiocarcinoma: A Single‐Center Retrospective Study DOI Creative Commons
Makiko Urabe, Takuo Yamai, Kenji Ikezawa

et al.

Liver international communications., Journal Year: 2025, Volume and Issue: 6(2)

Published: April 28, 2025

ABSTRACT The clinical utility of comprehensive genomic profiling (CGP) in intrahepatic cholangiocarcinoma (ICC) remains to be fully elucidated. This study analysed CGP test results and evaluated treatment outcomes with fibroblast growth factor receptor (FGFR) inhibitors patients ICC, aiming assess their efficacy cases specific FGFR alterations explore how can inform personalised strategies. We retrospectively reviewed data from 52 pathologically confirmed advanced who successfully underwent the at a Japanese cancer referral centre between November 2019 March 2023. median patient age was 67 years. identified one (1.9%) high tumour mutation burden (TMB) revealed therapeutically relevant oncogenic driver gene 32.7% cases. most frequently detected were FGFR2 (15.4%) isocitrate dehydrogenase 1 mutations (9.6%). Based on results, nine patients—eight fusions or rearrangements TMB—were eligible for approved targeted therapies. Among these, four treated pemigatinib achieved stable disease, duration 244 days. Notably, an fusion responded following futibatinib failure. test, when implemented timely manner, serve as valuable tool practice identifying novel therapeutic options ICC. Furthermore, sequential therapy may effective strategy managing rearrangements.

Language: Английский

Citations

0

Pharmacologic features, clinical applications, and drug safety evaluation of futibatinib in the treatment of biliary tract cancer (BTC) DOI
Angelo Pirozzi,

Celine Hoyek,

Naohiro Okano

et al.

Expert Opinion on Drug Safety, Journal Year: 2025, Volume and Issue: unknown, P. 1 - 8

Published: May 1, 2025

Futibatinib is a small, potent, covalent, irreversible fibroblast growth factor receptor (FGFR) 1-4 inhibitor that has been added as new standard of care for previously treated unresectable and/or advanced FGFR2 fusion/rearrangement-positive BTC. fusions/rearrangements play key role in BTC survival, proliferation, invasion, and development distant metastasis. The inhibition this pathway an important target the treatment article covers futibatinib refractory unresectable/advanced BTC, its mechanism action, pharmacodynamic/pharmacokinetic data with focus on safety profile. Data are based published clinical trials, pooled analysis, retrospective studies indexed PubMed (2010-2024). FDA EMA approved patients fusions/rearrangements. Ongoing drug strategies centered designing fusion inhibitors able to overcome on-target off-target resistances coupled high selectivity spare most common treatment-related adverse events (hyperphosphatemia, stomatitis, alopecia, nail toxicity, skin reactions, eye toxicity).

Language: Английский

Citations

0

Durvalumab plus chemotherapy in advanced biliary tract cancer: 3-year overall survival update from the phase III TOPAZ-1 study DOI Creative Commons
Do‐Youn Oh, Aiwu Ruth He, Shukui Qin

et al.

Journal of Hepatology, Journal Year: 2025, Volume and Issue: unknown

Published: May 1, 2025

Language: Английский

Citations

0

How I Treat Advanced Biliary Cancer DOI
Arathi Mohan, Vaibhav Sahai

Advances in Oncology, Journal Year: 2025, Volume and Issue: unknown

Published: Feb. 1, 2025

Language: Английский

Citations

0