Neoadjuvant Chemoimmunotherapy for the Treatment of Locally Advanced Nasopharyngeal Carcinoma in Non-Endemic Regions: A Single-Arm Phase 2 Clinical Trial

Lingran Zhou, Zhenchao Tao, Yan Zhou, Liping Yang, Yangyang Zhang, Jian He, Bin Sun, Ru Wang, Jing Yang, Jin Gao Department of Radiation Oncology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui, People’s Republic of ChinaCorrespondence: Jin Gao, Department of Radiation Oncology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui, People’s Republic of China, Email gjfl2011@126.comBackground and Purpose: Nasopharyngeal carcinoma (NPC) is a malignancy with distinct geographical distribution and is rare in non-endemic regions. Despite significant improvements in NPC survival through chemoradiotherapy, recurrence and metastasis remain major clinical challenges, particularly in non-endemic areas where data on neoadjuvant immunochemotherapy are scarce. This study aimed to address this gap by evaluating the antitumor activity and safety of neoadjuvant gemcitabine–cisplatin plus camrelizumab in patients with locally advanced NPC in non-endemic regions.Materials and Methods: In this single-center, single-arm, phase 2 trial, patients with stage III–IVA NPC received chemotherapy (gemcitabine 1000 mg/m2 on days 1 and 8, plus cisplatin 80 mg/m2 on day 1) and camrelizumab 200 mg on day 1 for three cycles, followed by radiotherapy. Primary endpoints were objective response rate (ORR). Secondary endpoints included disease-free survival (DFS), overall survival (OS), and biomarker correlations in addition to the primary ORR.Results: Thirty-eight patients were enrolled and thirty-three completed three cycles of the neoadjuvant therapy, resulting in an ORR of 89.4% (34/38). The median follow-up duration was 18.5 months, and the 12-month disease-free survival rate was 94.7% (34/36). Six patients encountered grade 3 adverse effects: Two with nausea/vomiting, two with thrombocytopenia, one with leukopenia, and one with immune-related hepatitis. No grade 4 treatment-related events or deaths occurred.Conclusion: Neoadjuvant camrelizumab combined with chemotherapy demonstrated promising antitumor activity and acceptable safety in locally advanced NPC patients from non-endemic regions. However, further randomized studies are needed to confirm these findings and assess long-term survival benefits.Keywords: neoadjuvant chemoimmunotherapy, PD-1 inhibitor, camrelizumab, nasopharyngeal carcinoma, radiotherapy