FGFR2 testing in cholangiocarcinoma: translating molecular studies into clinical practice

Pathologica. 2023 Apr;115(2):71-82. doi: 10.32074/1591-951X-859. Epub 2023 Apr 5.

Abstract

Cholangiocarcinoma (CCA) is a heterogeneous group of neoplasms burdened by a dismal prognosis. Several studies have investigated the genomic profile of CCA and identified numerous druggable genetic alterations, including FGFR2 fusions/rearrangements. Approximately 5-7% of CCAs and 10-20% of intrahepatic iCCAs harbor FGFR2 fusions. With the recent advent of FGFR-targeting therapies into clinical practice, a standardization of molecular testing for FGFR2 alterations in CCA will be necessary. In this review, we describe the technical aspects and challenges related to FGFR2 testing in routine practice, focusing on the comparison between Next-Generation Sequencing (NGS) and FISH assays, the best timing to perform the test, and on the role of liquid biopsy.

Keywords: FGFR2; biomarkers; cholangiocarcinoma; precision medicine.

Publication types

  • Review

MeSH terms

  • Bile Duct Neoplasms* / diagnosis
  • Bile Duct Neoplasms* / drug therapy
  • Bile Duct Neoplasms* / genetics
  • Bile Ducts, Intrahepatic / pathology
  • Cholangiocarcinoma* / diagnosis
  • Cholangiocarcinoma* / drug therapy
  • Cholangiocarcinoma* / genetics
  • Humans
  • Mutation
  • Prognosis
  • Receptor, Fibroblast Growth Factor, Type 2 / genetics
  • Receptor, Fibroblast Growth Factor, Type 2 / therapeutic use

Substances

  • FGFR2 protein, human
  • Receptor, Fibroblast Growth Factor, Type 2