Neoadjuvant tislelizumab plus chemotherapy in locally advanced oral and oropharyngeal squamous cell carcinoma: A single-arm phase II clinical trial

Abstract

Background

This study aimed to evaluate the antitumor effect and safety of neoadjuvant chemotherapy plus tislelizumab (a programmed death-1 inhibitor) for the treatment of resectable locally advanced oral or oropharyngeal squamous cell carcinoma (LAOOPSCC).

Methods

Eligible patients with stage III-IV LAOOPSCC were treated with two cycles of tislelizumab (200 mg), albumin-bound paclitaxel (260 mg/m²), and cisplatin (60–75 mg/m²) with a three-week interval between each cycle; this treatment was followed by surgery and adjuvant radiotherapy or concurrent chemoradiotherapy. The primary endpoints were the major pathological response (MPR) rate and safety.

Results

Between March 2022 and June 2023, a total of 82 patients were eligible and completed two cycles of neoadjuvant therapy; 78 patients underwent imaging evaluation, and 73 underwent surgery and pathological response evaluation. Forty patients underwent de-escalation surgery for the primary tumor. Orbital floor resection was avoided in 1 patient, mandibulectomy was avoided in 5 patients, and near-total glossectomy or total glossectomy was avoided in 32 patients. An objective response rate of 67.9 % and an MPR rate of 60.3 % were achieved, with 34.2 % of patients achieving a pathological complete response. With a median follow-up time of 24 months, the two-year overall survival rate was 84.4 %, and the two-year event-free survival rate was 76.7 %. Treatment-related adverse events of grade 3 or 4 occurred in 11 patients (13.4 %) during the neoadjuvant therapy.

Conclusions

Neoadjuvant tislelizumab plus chemotherapy for LAOOPSCC achieved a high pathological response rate and favorable survival metrics with an acceptable safety profile.