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      REVIEW | New horizons for precision medicine in biliary tract cancers

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          Abstract

          Biliary tract cancers (BTCs), including cholangiocarcinoma and gallbladder cancer, are poor-prognosis and low-incidence cancers, although the incidence of intrahepatic cholangiocarcinoma is rising. A minority of patients presents with resectable disease; however, relapse rates are high; benefit from adjuvant capecitabine chemotherapy has been suggested. Cisplatin/gemcitabine combination chemotherapy has emerged as the reference first-line treatment regimen; there is no standard second-line therapy. Selected patients may be suitable for liver-directed therapy (e.g. radioembolization or external beam radiation); pending confirmation of benefit in randomized studies. Initial trials targeting the epithelial growth factor receptor and angiogenesis pathways have failed to deliver new treatments. Emerging data from next generation sequencing analyses have identified actionable mutations (e.g. FGFR fusion rearrangements and IDH-1 and -2 mutations) with several targeted drugs entering clinical development with encouraging results. The role of systemic therapies, including targeted therapies and immunotherapy for BTC is rapidly evolving and the subject of this review.

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          Author and article information

          Journal
          101561693
          39259
          Cancer Discov
          Cancer Discov
          Cancer discovery
          2159-8274
          2159-8290
          4 August 2017
          17 August 2017
          September 2017
          01 March 2018
          : 7
          : 9
          : 943-962
          Affiliations
          [1 ]Department of Medical Oncology, The Christie NHS Foundation Trust, Wilmslow Road, Manchester, M20 4BX, UK. Phone: +44 161-446-8106; Fax +44 161-446-3468
          [2 ]Institute of Cancer Sciences, University of Manchester, Wilmslow Road, Manchester, M20 4BX, UK. Phone: +44 161-446-8106; Fax +44 161-446-3468
          [3 ]Massachusetts General Hospital Cancer Center, Harvard Medical School, 55 Fruit Street, Boston, MA 02114, USA. Phone: +1 617-726-2000; Fax: +1 617-724-1137
          [4 ]Faculty of Medical, Biological and Human Sciences, University of Manchester, Rumford Street, Manchester, M13 9PL, Manchester, UK. Phone: +44 161-446-8106; Fax +44 161-4463-468
          Author notes
          [* ] Corresponding Authors | Juan W. Valle: Department of Medical Oncology, The Christie NHS Foundation Trust, Wilmslow Road, Manchester, M20 4BX, UK. Phone: +44 161-446-8106; Fax +44 161-446-3468. juan.valle@ 123456christie.nhs.uk . Andrew X. Zhu: Massachusetts General Hospital Cancer Center, Harvard Medical School, 55 Fruit Street, Boston, MA 02114, USA. Phone: +1 617-726-2000; Fax: +1 617-724-1137. azhu@ 123456mgh.harvard.edu
          Article
          PMC5586506 PMC5586506 5586506 nihpa893401
          10.1158/2159-8290.CD-17-0245
          5586506
          28818953
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