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

[BACKGROUND 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/m on days 1 and 8, plus cisplatin 80 mg/m 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.