Systemic therapies for intrahepatic cholangiocarcinoma

Robin Kate Kelley, John Bridgewater, Gregory J. Gores, Andrew X. Zhu

Research output: Contribution to journalReview articlepeer-review

43 Scopus citations


Intrahepatic cholangiocarcinoma (iCCA) is a highly lethal hepatobiliary neoplasm whose incidence is increasing. Largely neglected for decades as a rare malignancy and frequently misdiagnosed as carcinoma of unknown primary, considerable clinical and investigative attention has recently been focused on iCCA worldwide. The established standard of care includes first-line (gemcitabine and cisplatin), second-line (FOLFOX) and adjuvant (capecitabine) systemic chemotherapy. Compared to hepatocellular carcinoma, iCCA is genetically distinct with several targetable genetic aberrations identified to date. Indeed, FGFR2 and NTRK fusions, and IDH1 and BRAF targetable mutations have been comprehensively characterised and clinical data is emerging on targeting these oncogenic drivers pharmacologically. Also, the role of immunotherapy has been examined and is an area of intense investigation. Herein, in a timely and topical manner, we will review these advances and highlight future directions of research.

Original languageEnglish (US)
Pages (from-to)353-363
Number of pages11
JournalJournal of hepatology
Issue number2
StatePublished - Feb 2020


  • Adjuvant therapy
  • Checkpoint inhibitors
  • FGFR
  • IDH

ASJC Scopus subject areas

  • Hepatology


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