Safety and Treatment Outcomes of Nivolumab for the Treatment of Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma: Retrospective Multicenter Cohort Study

SIMPLE SUMMARY: Nivolumab is an anti-PD-1 monoclonal antibody that has been approved in the management of recurrent/metastatic head and neck squamous cell carcinoma (HNSCC) with evidence of progression within 6 months of platinum-based chemotherapy. Most of the evidence regarding the use of nivolumab in this setting is provided within clinical trials with strict inclusion criteria. This multicenter retrospective cohort study evaluates the real-world outcomes of nivolumab use in four major cancer centers in England. This study demonstrates similar progression free survival and overall survival as that seen in the CHECKMATE-141 study. In addition, it demonstrates that nivolumab tends to be well tolerated, with only a 15% rate of immune-related toxicity. Our findings support the findings of other real-world studies that demonstrate improved progression free survival and potentially overall survival for those who have immune-related toxicity. ABSTRACT: Nivolumab is an anti-PD-1 monoclonal antibody currently used as immunotherapy for patients with recurrent/metastatic head and neck squamous cell carcinoma (HNSCC) with evidence of disease progression after platinum-based chemotherapy. This study evaluates real-world safety and treatment outcomes of non-trial nivolumab use. A retrospective multicenter cohort study of patients with recurrent/metastatic HNSCC treated with nivolumab between January 2017 and March 2020 was performed. Overall, 123 patients were included. The median age was 64 years, the majority of patients were male (80.5%) and had a smoking history (69.9%). Primary outcomes included overall response rate (ORR) of 19.3%, median progression-free survival (PFS) of 3.9 months, 1-year PFS rate of 16.8%, a median overall survival (OS) of 6.5 months and 1-year OS rate of 28.6%. These results are comparable to the CHECKMATE-141 study. Of 27 patients who had PD-L1 status tested, positive PD-L1 status did not significantly affect PFS (p = 0.86) or OS (p = 0.84). Nivolumab was well tolerated with only 15.1% experiencing immune-related toxicities (IRT) and only 6.7% of patients stopping due to toxicity. The occurrence of IRT appeared to significantly affect PFS (p = 0.01) but not OS (p = 0.07). Nivolumab in recurrent/metastatic HNSCC is well tolerated and may be more efficacious in patients who develop IRT.