Neoadjuvant GOLP in Resectable High-Risk Intrahepatic Cholangiocarcinoma

作者：Guo-Ming Shi, Xiaoyong Huang, Fei Liang, X Liang, Rui Dong, Qing-Hai Ye, Qi Gao, Yuan Ji, Zhengping Yu, Wenlong Zhai, Jia‐Cheng Lu, Xiao-Wu Li, Fu-Bao Liu, Kui Wang, Wei Yang, Jialin Zhang, Zhiqiang Hu, Shuang-Jian Qiu, Xiao-Ying Wang, Xin-Rong Yang, HUI-CHUAN SUN, Y Shi, Zhen‐Bin Ding, Weiren Liu, Xiaowu Huang, Cenying Huang, Ying-Hao Shen, Jian Sun, Yi-Feng He, Yuan‐Fei Peng, Yang Xu, Jian-Jun Zou, Jie Zhou, Jia Fan · 发表于：New England Journal of Medicine · 年份：2026 · DOI：10.1056/nejmoa2513918 · 被引用次数：14 · 研究领域：Cholangiocarcinoma and Gallbladder Cancer Studies、Gallbladder and Bile Duct Disorders、Bladder and Urothelial Cancer Treatments

BACKGROUND: No neoadjuvant treatment has been considered to be standard therapy for patients with resectable intrahepatic cholangiocarcinoma with high-risk factors for recurrence. The GOLP regimen (gemcitabine-oxaliplatin, lenvatinib, and an anti-programmed death 1 antibody) has shown promising efficacy with a manageable safety profile in advanced intrahepatic cholangiocarcinoma and biliary tract cancer. METHODS: In a phase 2-3 trial, we randomly assigned, in a 1:1 ratio, patients with resectable high-risk intrahepatic cholangiocarcinoma to the neoadjuvant group (intravenous gemcitabine-oxaliplatin plus toripalimab every 3 weeks for three cycles and oral lenvatinib once daily for 9 weeks, followed by curative resection) or the control group (curative resection and no neoadjuvant treatment). All patients received adjuvant capecitabine for eight cycles after surgery. The primary end point was event-free survival. Secondary end points included overall survival and safety. RESULTS: A total of 178 patients underwent randomization (88 patients to the neoadjuvant group and 90 to the control group). At the interim analysis at a median follow-up of 16.9 months, the median event-free survival was significantly longer in the neoadjuvant group (18.0 months; 95% confidence interval [CI], 13.8 to 27.6) than in the control group (8.7 months; 95% CI, 7.2 to 12.4) (P<0.001). Overall survival at 24 months was 79% (95% CI, 70 to 90) in the neoadjuvant group and 61% (95% CI, 50 to 75) in the con...