Efficacy and safety of neoadjuvant toripalimab plus chemotherapy in localized deficient mismatch repair/microsatellite instability-high gastric or esophagogastric junction adenocarcinoma (NICE): a multicentre, single-arm, exploratory phase 2 study

作者：Liying Zhao, Hao Liu, Yu Jiang, Shuqiang Yuan, Huayuan Liang, Wei Wang, Junliang Jiang, Lina Yu, Liang Li, Chen Zhao, Xinhua Chen, Xuefeng Zhong, Yating Zheng, Fengping Li, Tian Lin, Mingli Zhao, Tao Chen, Hao Chen, Yanfeng Hu, Guoxin Li · 发表于：EClinicalMedicine · 年份：2025 · DOI：10.1016/j.eclinm.2025.103421 · 被引用次数：8 · 研究领域：Esophageal Cancer Research and Treatment、Gastric Cancer Management and Outcomes、Cancer Immunotherapy and Biomarkers

Background In locally advanced gastric or gastroesophageal junction adenocarcinoma (GC/EGJC), deficient mismatch repair/microsatellite instability-high (dMMR/MSI-H) tumors exhibit high responsiveness to immunotherapy. The synergistic efficacy of neoadjuvant immunotherapy combined with chemotherapy in dMMR/MSI-H GC/EGJC remains uncertain. Methods The NICE trial is a multicentre, single-arm, exploratory phase 2 study conducted at six hospitals in China, evaluating the safety and efficacy of toripalimab in combination with CapeOX as perioperative therapy for locally advanced GC/EGJC across three biomarker-defined cohorts. This report presents findings from cohort C. Eligible patients were aged 18–75 years with histologically or cytologically confirmed GC/EGJC, confirmed dMMR/MSI-H status, and clinically staged as cT3–4aNxM0 or cT2N + M0 (AJCC 8th edition) based on contrast-enhanced CT or MRI, upper endoscopy, diagnostic laparoscopy, and peritoneal lavage cytology. Patients received four cycles of neoadjuvant toripalimab (240 mg IV every 3 weeks) plus CapeOX (capecitabine 1000 mg/m 2 orally twice daily on Days 1–14 and oxaliplatin 130 mg/m 2 IV on Day 1), followed by curative-intent surgery and up to four cycles of the same regimen as adjuvant therapy. The primary endpoint was the major pathological response (MPR) rate, defined as ≤10% residual viable tumor cells in the tumor specimen resected after neoadjuvant therapy. All patients who received at least one dose of treatment wer...