Phase 2 trial of neoadjuvant toripalimab plus chemotherapy for locally advanced nasopharyngeal carcinoma

A phase 2 study at Zhejiang Cancer Hospital (Hangzhou, China) evaluated neoadjuvant immunochemotherapy with 3 cycles of toripalimab plus gemcitabine and cisplatin for patients with locally advanced nasopharyngeal carcinoma (NPC). The study enrolled 91 patients aged 18-65 years with newly diagnosed, stage III-IV A NPC. The primary endpoint was complete response (CR). Tumor response was assessed 1 week after the completion of neoadjuvant immunochemotherapy. At last follow-up, the median follow-up time was 24.3 months (IQR 13.0-30.6). Neoadjuvant immunochemotherapy resulted in CR in 30 patients (33.0%; 95% CI 26.4-47.0). The two-year locoregional recurrence-free survival rate was 97.1% (95% CI 93.3-100), the two-year distant metastasis-free survival rate was 94.8% (89.3-100), and the two-year overall survival rate was 98.2% (94.4-100). Grade 3 treatment-related adverse events occurred in 36 patients (39.6%) and grade 4 treatment-related adverse events occurred in 10 patients (11.0%). CD44-mediated regulation of Treg abundance was identified as a critical determinant of response to neoadjuvant immunochemotherapy. The authors conclude that neoadjuvant toripalimab plus chemotherapy achieved promising efficacy with manageable toxicity among patients with locally advanced NPC.