The treatment outcomes in patients with squamous cell carcinoma of the oral cavity remain poor. However, recent studies have suggested that neoadjuvant immunotherapy prior to surgery and combining PD-1 and CTLA-4 immune checkpoint inhibitors can result in positive outcomes. This study aims to evaluate the efficacy and safety of nivolumab alone and nivolumab plus ipilimumab in patients with oral cavity squamous cell carcinoma.

This phase 2, randomized clinical trial included a total of 29 patients with untreated squamous cell carcinoma of the oral cavity. The patients were randomly assigned to receive nivolumab only or nivolumab and ipilimumab. The primary outcome of the study was the safety, and volumetric response determined using bidirectional measurements.

Toxic effects at least related to study treatment were observed in 21 patients, with grade 3 and 4 adverse events being observed in 2 patients in the nivolumab group and 5 patients in the nivolumab-ipilimumab group. One patient died during the study; however, the cause of death was considered to be unrelated to the study treatment. The 1-year progression-free survival was 85%, and overall survival was 89%.

The research concluded that treatment with both nivolumab and nivolumab plus ipilimumab was effective before surgical resection in patients with oral cavity squamous cell carcinoma.