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Pan et al. Hepatoma Res 2024;10:3 Hepatoma Research
DOI: 10.20517/2394-5079.2023.44

Review Open Access

Mutation-based therapies for intrahepatic

cholangiocarcinoma: new options on the horizon

1,2
2
Si-yuan Pan 1,2,# , Yu-Hang Ye 1,2,# , Zheng-Jun Zhou , Jia Fan , Jian Zhou 1,2 , Shao-Lai Zhou 1,2
1
Department of Liver Surgery and Transplantation, Zhongshan Hospital, Fudan University, Shanghai 200032, China.
2
Liver Cancer Institute, Zhongshan Hospital, Fudan University, Shanghai 200032, China.
#
Authors contributed equally.
Correspondence to: Prof. Shao-Lai Zhou, Department of Liver Surgery and Transplantation, Liver Cancer Institute, Zhongshan
Hospital, Fudan University, 1609 Xie Tu Road, Shanghai 200032, China. E-mail: zhoushaolai99@sina.com
How to cite this article: Pan Sy, Ye YH, Zhou ZJ, Fan J, Zhou J, Zhou SL. Mutation-based therapies for intrahepatic
cholangiocarcinoma: new options on the horizon. Hepatoma Res 2024;10:3. https://dx.doi.org/10.20517/2394-5079.2023.44
Received: 12 May 2023 First Decision: 25 Sep 2023 Revised: 19 Dec 2023 Accepted: 3 Jan 2024 Published: 12 Jan 2024

Academic Editor: Hong-Ping Xia Copy Editor: Yanbing Bai Production Editor: Yanbing Bai

Abstract
Intrahepatic cholangiocarcinoma (ICC), a rare but rising global malignancy originating from the bile ducts, poses
significant challenges in terms of effective treatment and patient outcomes. While surgical excision remains the
curative option, its limited efficacy necessitates more therapeutic strategies, including systemic therapies. The
management of ICC involves a multidisciplinary approach, with treatment decisions guided by patient-specific and
tumor-specific factors. Gemcitabine-cisplatin (GEMCIS) chemotherapy has been a standard first-line therapy, but
recent advancements in immunotherapy, particularly the introduction of durvalumab, have provided new hope.
Additionally, gene mutation-based therapies, targeting fibroblast growth factor receptors (FGFRs), isocitrate
dehydrogenase-1 (IDH1), human epidermal growth factor receptor-2 (HER2), and B-RAF proto-oncogene (BRAF),
offer promising prospects for personalized treatment. High-throughput genomic profiling technologies have
facilitated the identification of actionable targets and the development of innovative therapeutic approaches. This
review summarizes the mutation-based therapies in ICC, including FDA-approved targeted drugs and ongoing
clinical trials, highlighting the evolving landscape of ICC treatment.

Keywords: Intrahepatic cholangiocarcinoma, mutation-based therapy, precision medicine

INTRODUCTION
Cholangiocarcinoma, known as bile duct cancer, is a malignancy originating from the epithelial cells lining

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