Efficacy, safety and single-cell analysis of neoadjuvant immunochemotherapy in locally advanced oral squamous cell carcinoma: a phase II trial

Abstract The clinical activity of neoadjuvant immunochemotherapy (NAIC) for treating locally advanced oral squamous cell carcinoma (LA-OSCC) remains uncertain. This single-arm, phase II trial (ChiCTR2200066119) tested 2 cycles of NAIC with camrelizumab plus nab-paclitaxel and cisplatin in LA-OSCC pa...

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Main Authors: Zhongzheng Xiang, Xiaoyuan Wei, Zhuoyuan Zhang, Yueyang Tang, Linyan Chen, Chenfeng Tan, Yuanyuan Zeng, Jun Wang, Guile Zhao, Zelei Dai, Mingmin He, Ningyue Xu, Chunjie Li, Yi Li, Lei Liu
Format: Article
Language:English
Published: Nature Portfolio 2025-04-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-59004-w
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Summary:Abstract The clinical activity of neoadjuvant immunochemotherapy (NAIC) for treating locally advanced oral squamous cell carcinoma (LA-OSCC) remains uncertain. This single-arm, phase II trial (ChiCTR2200066119) tested 2 cycles of NAIC with camrelizumab plus nab-paclitaxel and cisplatin in LA-OSCC patients. For primary endpoint, the major pathological response (MPR) rate was 69.0% (95% confidence interval (CI): 49.2%-84.7%). The treatment was well-tolerated, with only 2 patients (6.45%) having grade 3 or 4 treatment-related adverse events during neoadjuvant treatment. For secondary endpoints, the pathological complete response rate was 41.4% (95%CI: 23.5%-61.1%) and the objective response rate was 82.8% (24/29, 95%CI: 64.2%-94.2%). The 18-month overall survival and disease-free survival probabilities were 96.77% (95%CI: 79.23%-99.54%) and 85.71% (95%CI: 53.95%-96.22%), respectively. Exploratory analysis showed that patients with MPR exhibited higher density of baseline CD4_Tfh_CXCL13 cells, and increased density of tertiary lymphoid structures after NAIC. Baseline CD4_Tfh_CXCL13 cells might be potential predictive biomarker of efficacy. The interaction between CXCL13 on CD4_Tfh_CXCL13 cells and CXCR5 on B cells may play a role in treatment response. These findings suggest the potential of NAIC as a promising treatment for LA-OSCC and offer preliminary insights into responsive biomarkers.
ISSN:2041-1723