Toripalimab Plus Chemotherapy for Recurrent or Metastatic Nasopharyngeal Carcinoma

• DOI: 10.1001/jama.2023.20181
• Publication Date: 2023-11-28T16:01:43Z
• Authors: Hai-Qiang Mai; Qiu-Yan Chen; Dongping Chen; Chaosu Hu; Kunyu Yang; Jiyu Wen; Jingao Li; Yingrui Shi; Feng Jin; Ruilian Xu; Jianji Pan; Shenhong Qu; Ping Li; Chunhong Hu; Yi-Chun Liu; Yi Jiang; Xia He; Hung-Ming Wang; Wan-Teck Lim; Wangjun Liao; Xiaohui He; Xiaozhong Chen; Siyang Wang; Xianglin Yuan; Qi Li; Xiaoyan Lin; Shanghua Jing; Yanju Chen; Yin Lu; Ching-Yun Hsieh; Muh-Hwa Yang; Chia-Jui Yen; Jens Samol; Xianming Luo; Xiaojun Wang; Xiongwen Tang; Hui Feng; Sheng Yao; Patricia Keegan; Rui-Hua Xu
• Keywords: Progression-free survival; Clinical endpoint

Importance There are currently no therapies approved by the US Food and Drug Administration for nasopharyngeal carcinoma (NPC). Gemcitabine-cisplatin is current standard of care first-line treatment recurrent or metastatic NPC (RM-NPC). Objective To determine whether toripalimab in combination with gemcitabine-cisplatin will significantly improve progression-free survival overall as RM-NPC, compared alone. Design, Setting, Participants JUPITER-02 an international, multicenter, randomized, double-blind phase 3 study conducted NPC-endemic regions, including mainland China, Taiwan, Singapore. From November 10, 2018, to October 20, 2019, 289 patients RM-NPC prior systemic chemotherapy RM setting were enrolled from 35 participating centers. Interventions Patients randomized (1:1) receive (240 mg [n = 146]) placebo (n 143) up 6 cycles, followed maintenance until disease progression, intolerable toxicity, completion 2 years treatment. Main Outcome Progression-free assessed a blinded independent central review. Secondary end points included objective response rate, survival, investigator, duration response, safety. Results Among (median age, 46 [IQR, 38-53 years; 17% female), at final analysis, had longer than (median, 21.4 vs 8.2 months; HR, 0.52 [95% CI, 0.37-0.73]). With median follow-up 36.0 months, significant improvement was identified over (hazard ratio [HR], 0.63 0.45-0.89]; 2-sided P .008). The not reached group, while it 33.7 months group. A consistent effect on favoring toripalimab, found subgroups high low PD-L1 (programmed death–ligand 1) expression. incidence all adverse events, grade greater fatal events similar between groups. However, leading discontinuation (11.6% 4.9%), immune-related (54.1% 21.7%), (9.6% 1.4%) more frequent Conclusions Relevance addition provided statistically clinically meaningful benefits alone, manageable safety profile. These findings support use plus new this patient population. Trial Registration ClinicalTrials.gov Identifier: NCT03581786