Neoadjuvant presurgical PD-1 inhibition in oral cavity squamous cell carcinoma

Oral cavity squamous cell carcinoma (OCSCC) is a prevalent surgically treated subset of head and neck cancer with frequent recurrence and poor survival. Immunotherapy has demonstrated efficacy in recurrent/metastatic head and neck cancer. However, whether antitumor responses could be fostered by neoadjuvant presurgical immunotherapy remains unclear. Using a Simon’s two-stage design, we present results of a single-arm phase-II trial where 12 patients with stage II-IVA OCSCC received 3 to 4 biweekly doses of 3 mg/kg nivolumab followed by definitive surgical resection with curative intent. Presurgical nivolumab therapy in this cohort shows an overall response rate of 33% (n = 4 patients; 95% CI: 12%–53%). With a median follow up of 2.23 years, 10 out of 12 treated patients remain alive. Neoadjuvant nivolumab is safe, well-tolerated, and is not associated with delays in definitive surgical treatment in this study. This work demonstrates feasibility and safety for incorporation of nivolumab in the neoadjuvant setting for OCSCC (ClinicalTrials.gov: NCT03021993). [Display omitted] •Presurgical nivolumab in patients with resectable oral cancer has an ORR of 33%•10 out of 12 treated patients remain alive with a median follow up of 2.23 years•Neoadjuvant nivolumab is safe, well-tolerated, and not associated with treatment delays•This shows feasibility of nivolumab in the neoadjuvant setting for OCSCC (NCT03021993) Oral cavity squamous cell carcinoma is a prevalent subset of head and neck cancer with high recurrence rates and poor survival outcomes despite complex multimodality therapy. The current study by Knochelmann et al. demonstrates that presurgical nivolumab therapy has an overall response rate of 33% in this patient population.