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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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