Neoadjuvant immunotherapy with or without chemotherapy in locally advanced oral squamous cell carcinoma: Randomized, two-arm, phase 2 trial
Haiming Liu, Xuepeng Xiong, Zi‐Li Yu, Zhe Shao, Gaili Chen, Gaili Chen, Yu-Tong Liu, Xinxin Wang, Qiuyun Fu, Xiaoxia Cheng, Jing Li, Jiali Zhang, Bo Li, Hongyun Gong, Yahua Zhong, Wei Zhang, Jun Jia, Bing Liu, Gang Chen, Gang Chen
Abstract
Patients with locally advanced oral squamous cell carcinoma (OSCC) have poor outcomes with standard care. Neoadjuvant therapy is shown to be effective for these patients. In the randomized, two-arm, phase 2, non-comparative trial, we investigate the efficacy and safety of the neoadjuvant programmed cell death 1 (PD-1) inhibitor camrelizumab with or without docetaxel-cisplatin-5-fluorouracil (TPF) chemotherapy in patients with resectable locally advanced OSCC. Patients with stage III–IVA OSCC receive neoadjuvant therapy with three cycles of camrelizumab (arm Cam) with or without two cycles of TPF chemotherapy (arm Cam+TPF), followed by surgery and adjuvant therapy. Major pathological response (MPR) is achieved in both arm Cam (5/34, 14.7%) and arm Cam+TPF (26/34, 76.4%). With a median follow-up of 32 months, the 2-year event-free survival (EFS) rate of arm Cam and Cam+TPF is 52.9% and 91.2%, respectively. This work demonstrates feasibility and safety for immunochemotherapy in the neoadjuvant setting for OSCC. This study was registered at ClinicalTrials.gov ( NCT04649476 ). • Neoadjuvant camrelizumab plus TPF chemotherapy are feasible in locally advanced OSCC • The combination regimen is well tolerated and associated with durable survival benefits • Pathological partial response is a surrogate primary endpoint for neoadjuvant therapy • Non-responders to neoadjuvant immunochemotherapy necessitate close monitoring Patients with locally advanced oral squamous cell carcinoma often experience poor outcomes with standard care. Liu et al. conduct a randomized, two-arm, phase 2, non-comparative trial to evaluate the feasibility and safety profile of neoadjuvant camrelizumab, either alone or in combination with TPF chemotherapy, in patients with resectable locally advanced OSCC.