Retrospective Cohort Study
Copyright ©The Author(s) 2025. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Gastrointest Oncol. Apr 15, 2025; 17(4): 103776
Published online Apr 15, 2025. doi: 10.4251/wjgo.v17.i4.103776
Idarubicin-transarterial chemoembolization combined with gemcitabine plus cisplatin for unresectable intrahepatic cholangiocarcinoma
Cheng-Hao Zhao, Huan Liu, Tao Pan, Zhan-Wang Xiang, Lu-Wen Mu, Jun-Yang Luo, Chu-Ren Zhou, Ming-An Li, Ming-Ming Liu, Hu-Zheng Yan, Ming-Sheng Huang
Cheng-Hao Zhao, Huan Liu, Tao Pan, Zhan-Wang Xiang, Lu-Wen Mu, Jun-Yang Luo, Chu-Ren Zhou, Ming-An Li, Hu-Zheng Yan, Ming-Sheng Huang, Department of Interventional Radiology, Third Affiliated Hospital of Sun Yat-sen University, Guangzhou 510630, Guangdong Province, China
Ming-Ming Liu, Department of Radiology, Third Affiliated Hospital of Sun Yat-sen University, Guangzhou 510630, Guangdong Province, China
Co-first authors: Cheng-Hao Zhao and Huan Liu.
Co-corresponding authors: Hu-Zheng Yan and Ming-Sheng Huang.
Author contributions: Huang MS, Yan HZ, Zhao CH contributed to conceptualization; Liu H, Xiang ZW, Pan T contributed to data curation; Mu LW, Li MA, Liu MM contributed to formal analysis; Zhao CH, Yan HZ, Xiang ZW contributed to investigation; Zhao CH, Huang MS, Pan T contributed to methodology; Huang MS, Pan T, Xiang ZW contributed to project administration; Huang MS, Yan HZ, Zhou CR contributed to resources; Zhao CH, Liu H, Xiang ZW contributed to software; Huang MS, Liu H, Zhao CH contributed to supervision; Zhao CH, Mu LW, Luo JY contributed to writing original draft; Huang MS and Yan HZ contributed to writing, review and editing.
Supported by the Guangzhou Science and Technology Plan Project, No. 2023A04J0419; and National Natural Science Foundation Cultivation Project at the Third Affiliated Hospital of Sun Yat-sen University, No. 2022GZRPYQN04.
Institutional review board statement: This study has adhered to the respective ethical requirements for human and animal studies. In terms of human research, this retrospective clinical study has been approved by the Ethics Committee of the Third Affiliated Hospital of Sun Yat-sen University and has complied with the 1975 Helsinki Declaration (Ethics number: II2024-238-01).
Informed consent statement: As it was a retrospective study, informed consent was waived.
Conflict-of-interest statement: The authors declare that they have no conflict of interest.
STROBE statement: The authors have read the STROBE Statement—a checklist of items, and the manuscript was prepared and revised according to the STROBE Statement-a checklist of items.
Data sharing statement: The data supporting the findings of this study are available upon reasonable request from the corresponding author.
Open Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Ming-Sheng Huang, Department of Interventional Radiology, Third Affiliated Hospital of Sun Yat-sen University, No. 600 Tianhe Road, Guangzhou 510630, Guangdong Province, China. huangmsh@mail.sysu.edu.cn
Received: December 3, 2024
Revised: January 12, 2025
Accepted: February 7, 2025
Published online: April 15, 2025
Processing time: 116 Days and 2.1 Hours
Abstract
BACKGROUND

Intrahepatic cholangiocarcinoma (iCCA) is the second most common liver malignancy with poor prognosis and limited treatment options.

AIM

To identify the most effective drug for transarterial chemoembolization (TACE) in cholangiocarcinoma and evaluate the efficacy and safety of combining it with gemcitabine and cisplatin (GemCis) for unresectable iCCA.

METHODS

Cholangiocarcinoma cell lines (RBE, HuCC-T1) were treated with 10 chemotherapeutic drugs, and cytotoxicity was assessed by cell counting kit-8 assays. Tumor-bearing nude mice were treated with idarubicin or GemCis, and tumor growth was monitored. Clinical data from 85 iCCA patients were analyzed to evaluate the efficacy and safety of idarubicin-TACE combined with GemCis.

RESULTS

Idarubicin demonstrated the highest cytotoxicity, significantly outperforming GemCis, the standard first-line therapies. In tumor-bearing mouse models, idarubicin and GemCis treatments significantly slowed tumor growth, with idarubicin showing particularly pronounced effects on days 12 and 15 (P < 0.05). In retrospective analysis, the median overall survival (OS) and progression-free survival (PFS) in the combination therapy group were significantly longer than those in the GemCis alone group (median OS, 16.23 months vs 10.07 months, P = 0.042; median PFS, 7.73 months vs 6.30 months, P = 0.023). Additionally, major grade 3/4 adverse events (AEs) in the combination therapy group were abdominal pain (26.3% vs 6.5%, P = 0.049) and elevated transaminases (42.1% vs 12.9%, P = 0.038). Most AEs were mild to moderate and manageable.

CONCLUSION

Idarubicin demonstrated higher cytotoxicity than GemCis, significantly inhibiting tumor growth in tumor-bearing mouse models. Preliminary clinical results suggest that local idarubicin-TACE combined with GemCis may offer improved survival outcomes for iCCA patients with a manageable safety profile.

Keywords: Transarterial chemoembolization; Intrahepatic cholangiocarcinoma; Gemcitabine; Cisplatin; Idarubicin

Core Tip: Intrahepatic cholangiocarcinoma (iCCA) is a liver cancer with poor survival rates and limited treatments, prompting the need for novel therapeutic strategies. This study reveals that idarubicin demonstrates superior cytotoxicity compared to standard treatments and shows improved survival outcomes when combined with gemcitabine and cisplatin in both preclinical and clinical settings. The findings highlight the potential of idarubicin-transarterial chemoembolization as a promising therapeutic agent for unresectable iCCA, offering hope for better management of this challenging disease with a manageable safety profile.