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New systemic treatment options for advanced cholangiocarcinoma
Valentina Zanuso, Giulia Tesini, Elena Valenzi, Lorenza Rimassa
J Liver Cancer. 2024;24(2):155-170.   Published online August 8, 2024
DOI: https://doi.org/10.17998/jlc.2024.08.07
  • 33,367 Views
  • 583 Downloads
  • 14 Citations
AbstractAbstract PDF
Cholangiocarcinoma (CCA) is a rare and aggressive cancer, mostly diagnosed at advanced or metastatic stage, at which point systemic treatment represents the only therapeutic option. Chemotherapy has been the backbone of advanced CCA treatment. More recently, immunotherapy has changed the therapeutic landscape, as immune checkpoint inhibitors have yielded the first improvement in survival and currently, the addition of either durvalumab or pembrolizumab to standard of care cisplatin plus gemcitabine represents the new first-line treatment option. However, the use of immunotherapy in subsequent lines has not demonstrated its efficacy and therefore, it is not approved, except for pembrolizumab in the selected microsatellite instability-high population. In addition, advances in comprehensive genomic profiling have led to the identification of targetable genetic alterations, such as isocitrate dehydrogenase 1 (IDH1), fibroblast growth factor receptor 2 (FGFR2), human epidermal growth factor receptor 2 (HER2), proto-oncogene B-Raf (BRAF), neurotrophic tropomyosin receptor kinase (NTRK), rearranged during transfection (RET), Kirsten rat sarcoma virus (KRAS), and mouse double minute 2 homolog (MDM2), thus favoring the development of a precision medicine approach in previously treated patients. Despite these advances, the use of molecularly driven agents is limited to a subgroup of patients. This review aims to provide an overview of the newly approved systemic therapies, the ongoing studies, and future research challenges in advanced CCA management.

Citations

Citations to this article as recorded by  
  • Genomic and transcriptomic signatures of sequential carcinogenesis from papillary neoplasm to biliary tract cancer
    Taek Chung, Seungho Oh, Jeongsoo Won, Jiho Park, Jeong Eun Yoo, Ho Kyoung Hwang, Gi Hong Choi, Chang Moo Kang, Dai Hoon Han, Sangwoo Kim, Young Nyun Park
    Journal of Hepatology.2025; 83(1): 119.     CrossRef
  • A concise review of updated global guidelines for the management of hepatocellular carcinoma: 2017-2024
    Hyunjae Shin, Su Jong Yu
    Journal of Liver Cancer.2025; 25(1): 19.     CrossRef
  • Modulating Wnt/β-catenin pathway activity to enhance chemosensitivity in cholangiocarcinoma
    Kevin Delgado-Calvo, Luke Boulter, Oscar Briz, Aleksandra Rozyczko, Paula Olaizola, Jose J.G. Marin, Rocio I.R. Macias, Elisa Lozano
    Biomedicine & Pharmacotherapy.2025; 188: 118225.     CrossRef
  • Regorafenib plus modified gemcitabine-oxaliplatin in patients with advanced biliary tract cancer. The randomized phase Ib/II BREGO study
    Jean-Frédéric Blanc, Mohamed Bouattour, Ludovic Gauthier, Emmanuel Deshayes, Sophie Guillemard, Yann Touchefeu, Fabienne Portales, Christophe Borg, Lobna Harguem, Rosine Guimbaud, Laurent Mineur, Marc Ychou, Thibault Mazard, Eric Assenat
    The Oncologist.2025;[Epub]     CrossRef
  • The new era of cholangiocarcinoma treatment: application of nano-based drug delivery systems
    Paweena Dana, Prattana Tanyapanyachon, Saksorn Klibaim, Monthira Rattanatayarom, Walailuk Chonniyom, Nattika Saengkrit
    Hepatoma Research.2025;[Epub]     CrossRef
  • Diagnostic value of quantitative DWI and IVIM parameters in differentiating intrahepatic cholangiocarcinoma and hepatocellular carcinoma: a systematic review and meta-analysis
    Saeed Mohammadzadeh, Alisa Mohebbi, Mehrad Zare, Faeze Salahshour, Afshin Mohammadi
    Abdominal Radiology.2025;[Epub]     CrossRef
  • Advanced cholangiocarcinoma with human epidermal growth factor receptor 2 (HER2) amplification treated with Trastuzumab deruxtecan (T-DXd): A case report
    Xiaohui Bao, Zhi Chen, Jin Xiong, Zhenzhou Yang, Ni Zhang
    Medicine.2025; 104(35): e44094.     CrossRef
  • Validation of prognostic models for predicting postsurgical outcomes in intrahepatic cholangiocarcinoma patients using a multicenter cohort
    Dong Hwan Kim, Sang Hyun Choi, Sehee Kim, Woohyung Lee, Hyung-Don Kim, Hyungjin Rhee, Eun-Suk Cho, Suk-Keu Yeom, Sumi Park, Seung Soo Lee, Mi-Suk Park
    International Journal of Surgery.2025; 111(10): 7032.     CrossRef
  • The Antibody–Drug Conjugate Sacituzumab Govitecan (IMMU-132) Represents a Potential Novel Therapeutic Strategy in Cholangiocarcinoma
    Racha Hosni, Niklas Klümper, Christine Sanders, Sana Hosni, Vittorio Branchi, Alexander Semaan, Abdullah Alajati, Natalie Pelusi, Susanna S. Ng, Damian J. Ralser, Saif-Eldin Abedellatif, Hanno Matthaei, Jörg Kalff, Jasmitha Boovadira Poonacha, Veronika Lu
    Molecular Cancer Therapeutics.2025; 24(11): 1775.     CrossRef
  • Molecular Mechanisms and Therapeutic Perspectives of Gut Microbiota, Autophagy, and Apoptosis in Cholangiocarcinoma Pathophysiology
    Viviana A. Ruiz-Pozo, Santiago Cadena-Ullauri, Patricia Guevara-Ramírez, Rafael Tamayo-Trujillo, Elius Paz-Cruz, Alejandro Cabrera-Andrade, Ana Karina Zambrano
    International Journal of Molecular Sciences.2025; 26(20): 9949.     CrossRef
  • Beyond futility: The history and potential of liver transplantation in cholangiocarcinoma
    Lynn Affarah, Sreelakshmi Kotha, Philip Berry
    World Journal of Transplantation.2025;[Epub]     CrossRef
  • Is 26S proteasome non-ATPase regulatory subunit 6 a potential molecular target for intrahepatic cholangiocarcinoma?
    Yong-Zhi Zhuang, Li-Quan Tong, Xue-Ying Sun
    World Journal of Hepatology.2024; 16(11): 1219.     CrossRef
  • Imaging findings of intrahepatic cholangiocarcinoma for prognosis prediction and treatment decision-making: a narrative review
    Jun Gu Kang, Taek Chung, Dong Kyu Kim, Hyungjin Rhee
    The Ewha Medical Journal.2024;[Epub]     CrossRef
  • Resectability and survival outcome in real world practice of 720 cholangiocarcinoma patients: intrahepatic, perihilar and distal cholangiocarcinoma.
    Poowanai Sarkhampee, Weeris Ouransatien, Nithi Lertsawatvicha, Satsawat Chansitthichock, Paiwan Wattanarath
    World Journal of Surgical Oncology.2024;[Epub]     CrossRef
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A multidisciplinary approach with immunotherapies for advanced hepatocellular carcinoma
Yu Rim Lee
J Liver Cancer. 2023;23(2):316-329.   Published online September 22, 2023
DOI: https://doi.org/10.17998/jlc.2023.09.04
  • 7,149 Views
  • 170 Downloads
  • 11 Citations
AbstractAbstract PDF
Hepatocellular carcinoma (HCC) is a highly aggressive disease that is usually diagnosed at an advanced stage. Advanced HCC has limited treatment options and often has a poor prognosis. For the past decade, tyrosine kinase inhibitors have been the only treatments approved for advanced HCC that have shown overall survival (OS) benefits; however, but their clinical efficacy has been limited. Recent trials have demonstrated promising advancements in survival outcomes through immunotherapy-based treatments, such as combinations of immune checkpoint inhibitors (ICIs) with other ICIs, antiangiogenic drugs, and locoregional therapies. The atezolizumab-bevacizumab and durvalumab-tremelimumab (STRIDE) regimen has significantly improved survival rates as a first-line treatment and has become the new standard of care. Therefore, combined treatments for advanced HCC can result in better treatment outcomes owing to their synergistic effects, which requires a multidisciplinary approach. Ongoing studies are examining other therapeutic innovations that can improve disease control and OS rates. Despite improvements in the treatment of advanced HCC, further studies on the optimal treatment selection and sequences, biomarker identification, combination approaches with other therapies, and development of novel immunotherapy agents are required. This review presents the current treatment options and clinical data of the ICI-based combination immunotherapies for advanced HCC from a multidisciplinary perspective.

Citations

Citations to this article as recorded by  
  • Multidisciplinary treatment strategies for the assessment of immune, coagulation, and biomarker responses after transarterial chemoembolization for hepatocellular carcinoma
    Tian Song, Kan-Hua Wu, Hao Yang, Wen-Li Xie, Lan Shen
    World Journal of Gastrointestinal Surgery.2025;[Epub]     CrossRef
  • Reduced-Dose or Discontinuation of Bevacizumab Might Be Considered after Variceal Bleeding in Patients with Hepatocellular Carcinoma Receiving Atezolizumab/Bevacizumab: Case Reports
    Kyeong-Min Yeom, Young-Gi Song, Jeong-Ju Yoo, Sang Gyune Kim, Young Seok Kim
    Medicina.2024; 60(1): 157.     CrossRef
  • Hepatocellular Carcinoma: Advances in Systemic Therapy
    Insija Ilyas Selene, Merve Ozen, Reema A. Patel
    Seminars in Interventional Radiology.2024; 41(01): 056.     CrossRef
  • Fatal intratumoral hemorrhage in a patient with hepatocellular carcinoma following successful treatment with atezolizumab/bevacizumab: A case report
    Kyeong-Hoon Park, Jeong-Ju Yoo, Sang Gyune Kim, Young Seok Kim
    World Journal of Clinical Cases.2024; 12(22): 5177.     CrossRef
  • Unravelling infiltrating T‐cell heterogeneity in kidney renal clear cell carcinoma: Integrative single‐cell and spatial transcriptomic profiling
    Haiqing Chen, Haoyuan Zuo, Jinbang Huang, Jie Liu, Lai Jiang, Chenglu Jiang, Shengke Zhang, Qingwen Hu, Haotian Lai, Bangchao Yin, Guanhu Yang, Gang Mai, Bo Li, Hao Chi
    Journal of Cellular and Molecular Medicine.2024;[Epub]     CrossRef
  • Atezolizumab-Induced Ulcerative Colitis in Patient with Hepatocellular Carcinoma: Case Report and Literature Review
    Hyuk Kim, Yoon E Shin, Hye-Jin Yoo, Jae-Young Kim, Jeong-Ju Yoo, Sang Gyune Kim, Young Seok Kim
    Medicina.2024; 60(9): 1422.     CrossRef
  • A Potential Pneumothorax Induced by Immune Checkpoint Inhibitors: A Case Report and Literature Review
    Yoon-E Shin, Hyuk Kim, Jeong-Ju Yoo, Sang Gyune Kim, Young Seok Kim
    Medicina.2024; 60(10): 1634.     CrossRef
  • Prediction of PD-L1 expression in unresectable hepatocellular carcinoma with gadoxetic acid-enhanced MRI
    Jun Gu Kang, Kyunghwa Han, Taek Chung, Hyungjin Rhee
    European Journal of Radiology.2024; 181: 111772.     CrossRef
  • Advances in Understanding Hepatocellular Carcinoma Vasculature: Implications for Diagnosis, Prognostication, and Treatment
    Hyungjin Rhee, Young Nyun Park, Jin-Young Choi
    Korean Journal of Radiology.2024; 25(10): 887.     CrossRef
  • Current perspectives on the pharmacological treatment of advanced hepatocellular carcinoma: a narrative review
    Hye-Jin Yoo, Jeong-Ju Yoo, Sang Gyune Kim, Young Seok Kim
    The Ewha Medical Journal.2024;[Epub]     CrossRef
  • Improved survival with second-line hepatic arterial infusion chemotherapy after atezolizumab-bevacizumab failure in hepatocellular carcinoma
    Ji Yeon Lee, Jaejun Lee, Suho Kim, Jae-sung Yoo, Ji Hoon Kim, Keungmo Yang, Ji Won Han, Jeong Won Jang, Jong Yong Choi, Seung Kew Yoon, Ho Jong Chun, Jung Suk Oh, Pil Soo Sung
    Frontiers in Oncology.2024;[Epub]     CrossRef
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Systemic Treatment of Advanced or Metastatic Hepatocellular Carcinoma
Joong-Won Park, Hark Kyun Kim
Journal of the Korean Liver Cancer Study Group. 2002;2(1):1-7.   Published online July 31, 2002
  • 943 Views
  • 5 Downloads
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