Hu Yue, Jiang Xiong-Ying, Cai Xi, Chen Song, Chen Qi-Feng, Yi Jun-Zhe, Zhong Sui-Xing, Wang Jiong-Liang, Xu Jie, Tan Gen-Jun, Lyu Ning, Zhao Ming
Department of Minimally Invasive Interventional Therapy, Liver Cancer Study and Service Group, Sun Yat-sen University Cancer Center, Guangzhou, China.
State Key Laboratory of Oncology in South China, Sun Yat-sen University Cancer Center, Guangzhou, China.
J Gastrointest Oncol. 2025 Feb 28;16(1):209-225. doi: 10.21037/jgo-24-552. Epub 2025 Feb 26.
Given the limited efficacy of current first-line therapies, there is an urgent need to develop novel treatment strategies to improve the prognosis of patients with unresectable intrahepatic cholangiocarcinoma (uICC). This study aimed to evaluate the efficacy and safety of hepatic arterial infusion chemotherapy (HAIC) with infusional fluorouracil, leucovorin, and oxaliplatin (FOLFOX) regimens (HAIC-FO) plus anti-programmed death-(ligand) 1 immunotherapy [αPD-(L)1] antibody [HAIC+αPD-(L)1] compared to systemic chemotherapy (SYS) plus αPD-(L)1 antibody [SYS+αPD-(L)1] as a first-line treatment for patients with uICC.
In this retrospective study, treatment-naive uICC patients who were treated with HAIC+αPD-(L)1 or SYS+αPD-(L)1 were included. The clinical characteristics, therapeutic outcomes, and adverse events (AEs) of the patients in the two groups were compared. Propensity score matching (PSM) was performed to minimize biases between groups.
From January 2019 to January 2023, a total of 182 patients were enrolled; 147 patients were included in the HAIC+αPD-(L)1 group and 35 patients were included in the SYS+αPD-(L)1 group. After PSM, 61 and 26 patients were included in the HAIC+αPD-(L)1 and SYS+αPD-(L)1 groups, respectively. The HAIC+αPD-(L)1 group had longer median overall survival (mOS), median progression-free survival (mPFS), and median intrahepatic PFS (mIPFS) than did the SYS+αPD-(L)1 group (mOS: 14.5 . 10.5 months, P=0.02; mPFS: 10.4 . 6.4 months, P=0.02; mIPFS: 11.4 . 6.5 months, P<0.001). The overall incidence of AEs was comparable between the two groups, but the HAIC+αPD-(L)1 group had a lower incidence of grade 3-4 AEs related to anemia, leukopenia, weight loss, and fatigue.
HAIC+αPD-(L)1 had acceptable toxic effects and might improve outcomes compared to SYS+αPD-(L)1 as a first-line treatment for patients with uICC.
鉴于目前一线治疗的疗效有限，迫切需要开发新的治疗策略以改善不可切除肝内胆管癌（uICC）患者的预后。本研究旨在评估肝动脉灌注化疗（HAIC）联合氟尿嘧啶、亚叶酸钙和奥沙利铂（FOLFOX）方案（HAIC-FO）加抗程序性死亡-（配体）1免疫疗法[αPD-（L）1]抗体[HAIC+αPD-（L）1]与全身化疗（SYS）加αPD-（L）1抗体[SYS+αPD-（L）1]作为uICC患者一线治疗的疗效和安全性。
在这项回顾性研究中，纳入了接受HAIC+αPD-（L）1或SYS+αPD-（L）1治疗的初治uICC患者。比较两组患者的临床特征、治疗结果和不良事件（AE）。进行倾向评分匹配（PSM）以尽量减少组间偏差。
2019年1月至2023年1月，共纳入182例患者；HAIC+αPD-（L）1组纳入147例患者，SYS+αPD-（L）1组纳入35例患者。PSM后，HAIC+αPD-（L）1组和SYS+αPD-（L）1组分别纳入61例和26例患者。HAIC+αPD-（L）1组的中位总生存期（mOS）、中位无进展生存期（mPFS）和中位肝内无进展生存期（mIPFS）均长于SYS+αPD-（L）1组（mOS：14.5对10.5个月，P=0.02；mPFS：10.4对6.4个月，P=0.02；mIPFS：11.4对6.5个月，P<0.001）。两组AE的总发生率相当，但HAIC+αPD-（L）1组与贫血、白细胞减少、体重减轻和疲劳相关的3-4级AE发生率较低。
作为uICC患者的一线治疗，与SYS+αPD-（L）1相比，HAIC+αPD-（L）1具有可接受的毒性作用，可能改善治疗结果。
