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Alqahtani et al. Hepatoma Res 2020;6:70                          Hepatoma Research
               DOI: 10.20517/2394-5079.2020.65




               Review                                                                        Open Access


               Systemic therapy for advanced cholangiocarcinoma:
               new options on the horizon



               Saleh A. Alqahtani , Massimo Colombo 3
                               1,2
               1 Liver Transplant Center, King Faisal Specialist Hospital & Research Center, Riyadh 12713, Saudi Arabia.
               2 Division of Gastroenterology and Hepatology, Johns Hopkins University, Baltimore, MD 21287, USA.
               3 Liver Center, San Raffaele Hospital, Milan 20132, Italy.

               Correspondence to: Dr. Saleh A. Alqahtani, Division of Gastroenterology and Hepatology, Johns Hopkins University, 600 North
               Wolfe St, Blalock Suite 412, Baltimore, MD 21287, USA. E-mail: salqaht1@jhmi.edu
               How to cite this article: Alqahtani SA, Colombo M. Systemic therapy for advanced cholangiocarcinoma: new options on the
               horizon. Hepatoma Res 2020;6:70. http://dx.doi.org/10.20517/2394-5079.2020.65
               Received: 28 Jun 2020    First Decision: 2 Sep 2020    Revised: 3 Sep 2020    Accepted: 9 Sep 2020    Published: 12 Oct 2020

               Academic Editor: Shu-Kui Qin    Copy Editor: Cai-Hong Wang    Production Editor: Jing Yu



               Abstract
 Received:     First Decision:     Revised:     Accepted:    Published:
               Patients with unresectable cholangiocarcinoma (CCA) face a poor prognosis, and there are few effective
 Science Editor:     Copy Editor:     Production Editor: Jing Yu   treatment options for the disease. The standard of care for patients with locally advanced or metastatic CCA is
               chemotherapy with a gemcitabine-based doublet. Unfortunately, the clinical benefit obtained with these regimens
               is modest, with a median overall survival of about one year. For CCA that is chemotherapy-refractory or recurs
               after first-line chemotherapy, the treatment options are even more limited, and no relevant randomized controlled
               data are available. In recent years, molecular profiling has shed light on the molecular basis of CCA and identified
               subgroups of patients that might benefit from a personalized treatment approach. These efforts resulted in the
               recent FDA approval of the fibroblast growth factor receptor (FGFR) inhibitor, pemigatinib, as a second-line
               treatment for patients with advanced CCA harboring an FGFR2-fusion or rearrangement. Several other targeted
               agents also are under evaluation in patients with CCA, of which the isocitrate dehydrogenase inhibitor has had
               the most promising results. Finally, immunotherapy is being explored as a new treatment approach for advanced
               CCA patients; indeed, the immune checkpoint inhibitor pembrolizumab can already be used to treat CCAs that are
               mismatch repair deficient. This review is a comprehensive overview of the treatment options for CCA and offers a
               glimpse into what the future could hold for these patients.

               Keywords: Cholangiocarcinoma, fibroblast growth factor receptor inhibitor, isocitrate dehydrogenase inhibitor,
               immune checkpoint inhibitor




                           © The Author(s) 2020. Open Access This article is licensed under a Creative Commons Attribution 4.0
                           International License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted use,
                sharing, adaptation, distribution and reproduction in any medium or format, for any purpose, even commercially, as long
                as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license,
                and indicate if changes were made.


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