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Targeted Therapies in Advanced Cholangiocarcinoma: A Focus on FGFR Inhibitors

Alessandro Rizzo1

  • 1Department of Experimental, Diagnostic and Specialty Medicine, S. Orsola-Malpighi University Hospital, 40138 Bologna, Italy.

Medicina (Kaunas, Lithuania)
|June 2, 2021
PubMed

Insights

Novel therapies, including fibroblast growth factor receptor (FGFR) inhibitors, offer new hope for cholangiocarcinoma (CCA) patients. These targeted treatments, like pemigatinib, are showing promise for advanced CCA with specific genetic mutations.

Area of Science:

  • Oncology
  • Molecular Biology
  • Pharmacology

Background:

  • Cholangiocarcinoma (CCA) is a challenging cancer with poor outcomes.
  • Recent advances have identified actionable mutations in about 50% of CCA cases.
  • Targeted therapies are emerging as a promising treatment strategy for CCA.

Purpose of the Study:

  • To review the current evidence on fibroblast growth factor receptor (FGFR) inhibitors in cholangiocarcinoma (CCA).
  • To discuss the development, challenges, and future directions of FGFR inhibitors for CCA treatment.

Main Methods:

  • Literature review of clinical trials and studies on FGFR inhibitors in CCA.
  • Analysis of efficacy and safety data for approved and investigational FGFR inhibitors.
  • Examination of genetic alterations, particularly FGFR2 fusions and rearrangements, in CCA patients.

Main Results:

  • FGFR inhibitors have demonstrated significant efficacy in pre-treated metastatic CCA patients with FGFR2 alterations.
  • Pemigatinib received FDA approval for CCA patients with FGFR2 fusions or rearrangements.
  • Ongoing research is exploring various FGFR inhibitors and combinations for CCA.

Conclusions:

  • FGFR inhibitors represent a significant advancement in personalized medicine for a subset of CCA patients.
  • Addressing the development, pitfalls, and challenges is crucial for optimizing their use.
  • Further research is needed to expand the application and improve outcomes with FGFR inhibitors in CCA.
Keywords:
FGFRcholangiocarcinomaintrahepatic cholangiocarcinomapemigatinibtargeted therapies

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