Neoadjuvant and adjuvant toripalimab for locoregionally advanced nasopharyngeal carcinoma: a randomised, single-centre, double-blind, placebo-controlled, phase 2 trial

作者：Sai-Lan Liu, Xiao-Yun Li, Jin-Hao Yang, Dong-Xiang Wen, Shanshan Guo, Li-Ting Liu, Yifu Li, Mei-Juan Luo, Si-Yi Xie, Yu-Jing Liang, Xue-Song Sun, Zhen-Chong Yang, Xiaofei Lv, Dong-Hua Luo, Ji-Bin Li, Qing Liu, Pan Wang, Ling Guo, Hao‐Yuan Mo, Rui Sun, Qi Yang, Kai‐Qi Lan, Guo-Dong Jia, Ru Li, Chong Zhao, Rui‐Hua Xu, Qiu-Yan Chen, Lin-Quan Tang, Hai‐Qiang Mai · 发表于：The Lancet Oncology · 年份：2024 · DOI：10.1016/s1470-2045(24)00504-7 · 被引用次数：63 · 研究领域：Head and Neck Cancer Studies、Viral-associated cancers and disorders、Head and Neck Surgical Oncology

BACKGROUND: Patients with locoregionally advanced nasopharyngeal carcinoma with a high pretreatment plasma concentration of Epstein-Barr virus (EBV) DNA remain at high risk for recurrence after concurrent chemoradiotherapy. This study aimed to compare the efficacy and safety of neoadjuvant-adjuvant treatment with the PD-1 inhibitor toripalimab and concurrent chemoradiotherapy versus placebo and concurrent chemoradiotherapy in patients with locoregionally advanced nasopharyngeal carcinoma. METHODS: intravenously) on days 1, 22, and 43 during intensity-modulated radiotherapy and adjuvant toripalimab (240 mg intravenously) or placebo once every 3 weeks for up to eight cycles. The primary endpoint was 2-year progression-free survival in the intention-to-treat population. This study was registered with ClinicalTrials.gov, NCT03925090, and is closed to enrolment; follow-up is ongoing. FINDINGS: Between Dec 6, 2019, and Dec 9, 2021, 150 patients were enrolled and randomly assigned to the toripalimab group (n=100) or placebo group (n=50). 115 (77%) patients were male and 35 (23%) were female. As of data cutoff (May 31, 2024), median follow-up for progression-free survival was 37·8 months (IQR 34·2-46·5) for the intention-to-treat population analyses. 2-year progression-free survival was higher in the toripalimab group (92·0% [95% CI 86·7-97·3]) than in the placebo group (74·0% [61·8-86·2]; stratified hazard ratio 0·40 [95% CI 0·18-0·89]; log-rank p=0·019). The most common grade 3 or ...