Targeted Therapies for Perihilar Cholangiocarcinoma

Simon Gray, Angela Lamarca, Julien Edeline, Heinz-josef Klümpen, Richard A. Hubner, Mairéad G. Mcnamara, Juan W. Valle

Research output: Contribution to journalArticlepeer-review


Perihilar cholangiocarcinoma (pCCA) is the anatomical sub-group of biliary tract cancer (BTC) arising between the second-order intrahepatic bile ducts and the cystic duct. Together with distal and intrahepatic cholangiocarcinoma (dCCA and iCCA; originating distal to, and proximal to this, respectively), gallbladder cancer (GBC) and ampulla of Vater carcinoma (AVC), these clini-copathologically and molecularly distinct entities comprise biliary tract cancer (BTC). Most pCCAs are unresectable at diagnosis, and for those with resectable disease, surgery is extensive, and recurrence is common. Therefore, the majority of patients with pCCA will require systemic treatment for advanced disease. The prognosis with cytotoxic chemotherapy remains poor, driving interest in therapies targeted to the molecular nature of a given patient’s cancer. In recent years, the search for efficacious targeted therapies has been fuelled both by whole-genome and epigenomic studies, looking to uncover the molecular landscape of CCA, and by specifically testing for aberrations where established therapies exist in other indications. This review aims to provide a focus on the current molecular characterisation of pCCA, targeted therapies applicable to pCCA, and future di-rections in applying personalised medicine to this difficult-to-treat malignancy.

Original languageEnglish
Article number1789
Pages (from-to)1789
Issue number7
Early online date31 Mar 2022
Publication statusPublished - 1 Apr 2022


  • biliary tract cancer
  • cholangiocarcinoma
  • extrahepatic
  • pCCA
  • perihilar
  • targeted therapy

Research Beacons, Institutes and Platforms

  • Manchester Cancer Research Centre


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