Precision approaches for cholangiocarcinoma: progress in clinical trials and beyond

Pedro Luiz Serrano Uson Junior, Mitesh J. Borad

Research output: Contribution to journalReview articlepeer-review


Introduction: Although a relatively uncommon tumor, the incidence of intrahepatic cholangiocarcinoma is rising globally. Unfortunately, most patients are diagnosed with locally advanced or metastatic disease with poor prognosis. Strategies targeting genomic alterations and incorporation of precision medicine will represent a new therapeutic avenue for these patients. Areas covered: In this review article, we addressed clinical trials in cholangiocarcinoma with FGFR, IDH, BRAF, and ErbB2 targeted therapies. We also reviewed mechanisms of resistance to precision medicine and possible future strategies to overcome clonal evolution. Articles selected for this review were based on reported studies indexed in PubMed (2010–2021). Expert Opinion: Pemigatinib, infigratinib and futibatinib could eventually be incorporated in the landscape of first-line systemic treatment for advanced cholangiocarcinoma with FGFR2 fusions or rearrangements after the ongoing phase III trials. Circulating tumor DNA could be used as a dynamic tool for evaluating mechanisms of resistance and prediction of response in patients treated with directed therapy.

Original languageEnglish (US)
Pages (from-to)125-131
Number of pages7
JournalExpert Opinion on Investigational Drugs
Issue number1
StatePublished - 2022


  • BRAF
  • Cholangiocarcinoma
  • FGFR2
  • HER2
  • IDH
  • biliary tract cancers
  • futibatinib
  • infigratinib
  • intrahepatic cholangiocarcinoma
  • pemigatinib
  • precision medicine

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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