Perioperative nivolumab and chemotherapy in locally advanced squamous cell carcinoma of the oesophagus: a randomized multicentre phase 2 study with circulating tumor DNA dynamics monitoring

作者：Heng Jiao, Siyun Lin, Jianmin Gu, Dongxian Jiang, Peng Cui, Zhiliang Huang, Yong Fang, Hao Wang, Lin Miao, Han Tang, Tian Jiang, Guang-Yi Lin, Shaoyuan Zhang, Hao Yin, Fei Liang, Jingshu Wang, Xuning Fan, Fujun Qiu, Yang Yang, Zhigang Li, Bin Li, Jia-Qing Xiang, Xuefeng Leng, Yongtao Han, Chun Li, Luoyan Ai, Yingyong Hou, Guoqiang Wang, Zhihong Zhang, Shangli Cai, Tianshu Liu, Jun Yin, Lijie Tan · 发表于：Molecular Cancer · 年份：2025 · DOI：10.1186/s12943-025-02332-8 · 被引用次数：13 · 研究领域：Esophageal Cancer Research and Treatment、Cancer Immunotherapy and Biomarkers、Cancer Cells and Metastasis

BACKGROUND: Although neoadjuvant chemotherapy and immunotherapy show promise in treating oesophageal squamous cell carcinoma (OSCC), long-term survival data are limited. This randomized, multicenter phase 2 study evaluated the efficacy of perioperative Nivolumab with chemotherapy, followed by surgery and adjuvant immunotherapy, in patients with locally advanced resectable OSCC, and explored the prognostic role of circulating tumor DNA (ctDNA) status. METHODS: In this trial, participants recruited from five centers were randomly assigned in a 2:1 ratio to receive either perioperative Nivolumab or a placebo in addition to chemotherapy (cisplatin and paclitaxel), followed by minimally invasive esophagectomy. For those who did not achieve a pathological complete response (pCR), adjuvant treatment with Nivolumab was administered. The main measure of success was the pCR rate, with secondary endpoints including the R0 resection rate, event-free survival, and overall survival. All outcomes and safety measures were assessed based on the intention-to-treat population. ctDNA levels were monitored as exploratory endpoints. RESULTS: Ninety patients were enrolled and randomized to Nivolumab or placebo plus chemotherapy. The pCR rate was slightly higher in the Nivolumab group (15%) compared to the control group (13.3%) (relative risk, 1.13; 95% CI, 0.38 to 3.36). No significant differences were observed in R0 resection rates (96.4% vs. 96.6%; P > 0.05). The median follow-up duration was 24....