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Targeted Therapies in Advanced Cholangiocarcinoma: A Focus on FGFR Inhibitors
Despite advanced diseases continuing to be associated with grim prognoses, the past decade has witnessed the advent of several novel treatment options for cholangiocarcinoma (CCA) patients. In fact, CCA has emerged as a heterogeneous group of malignancies harboring potentially druggable mutations in...
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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MDPI
2021
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8151905/ https://www.ncbi.nlm.nih.gov/pubmed/34066684 http://dx.doi.org/10.3390/medicina57050458 |
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author | Rizzo, Alessandro |
author_facet | Rizzo, Alessandro |
author_sort | Rizzo, Alessandro |
collection | PubMed |
description | Despite advanced diseases continuing to be associated with grim prognoses, the past decade has witnessed the advent of several novel treatment options for cholangiocarcinoma (CCA) patients. In fact, CCA has emerged as a heterogeneous group of malignancies harboring potentially druggable mutations in approximately 50% of cases, and thus, molecularly targeted therapies have been actively explored in this setting. Among these, fibroblast growth factor receptor (FGFR) inhibitors have reported important results, as witnessed by the FDA approval of pemigatinib in previously treated metastatic CCA patients harboring FGFR2 fusion or other rearrangements. Herein, we provide an overview of available evidence on FGFR inhibitors in CCA, especially focusing on the development, pitfalls and challenges of emerging treatments in this setting. |
format | Online Article Text |
id | pubmed-8151905 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-81519052021-05-27 Targeted Therapies in Advanced Cholangiocarcinoma: A Focus on FGFR Inhibitors Rizzo, Alessandro Medicina (Kaunas) Review Despite advanced diseases continuing to be associated with grim prognoses, the past decade has witnessed the advent of several novel treatment options for cholangiocarcinoma (CCA) patients. In fact, CCA has emerged as a heterogeneous group of malignancies harboring potentially druggable mutations in approximately 50% of cases, and thus, molecularly targeted therapies have been actively explored in this setting. Among these, fibroblast growth factor receptor (FGFR) inhibitors have reported important results, as witnessed by the FDA approval of pemigatinib in previously treated metastatic CCA patients harboring FGFR2 fusion or other rearrangements. Herein, we provide an overview of available evidence on FGFR inhibitors in CCA, especially focusing on the development, pitfalls and challenges of emerging treatments in this setting. MDPI 2021-05-08 /pmc/articles/PMC8151905/ /pubmed/34066684 http://dx.doi.org/10.3390/medicina57050458 Text en © 2021 by the author. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Rizzo, Alessandro Targeted Therapies in Advanced Cholangiocarcinoma: A Focus on FGFR Inhibitors |
title | Targeted Therapies in Advanced Cholangiocarcinoma: A Focus on FGFR Inhibitors |
title_full | Targeted Therapies in Advanced Cholangiocarcinoma: A Focus on FGFR Inhibitors |
title_fullStr | Targeted Therapies in Advanced Cholangiocarcinoma: A Focus on FGFR Inhibitors |
title_full_unstemmed | Targeted Therapies in Advanced Cholangiocarcinoma: A Focus on FGFR Inhibitors |
title_short | Targeted Therapies in Advanced Cholangiocarcinoma: A Focus on FGFR Inhibitors |
title_sort | targeted therapies in advanced cholangiocarcinoma: a focus on fgfr inhibitors |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8151905/ https://www.ncbi.nlm.nih.gov/pubmed/34066684 http://dx.doi.org/10.3390/medicina57050458 |
work_keys_str_mv | AT rizzoalessandro targetedtherapiesinadvancedcholangiocarcinomaafocusonfgfrinhibitors |