Ferris RL Blumenschein G Fayette J Guigay J Colevas AD Licitra L Harrington K Kasper S Vokes EE Even C Worden F Saba NF Iglesias Docampo LC Haddad R Rordorf T Kiyota N Tahara M Monga M Lynch M Geese WJ Kopit J Shaw JW Gillison ML 2016 BACKGROUND Patients with recurrent or metastatic squamous-cell carcinoma of the head and neck after platinum chemotherapy have a very poor prognosis and limited therapeutic options. Nivolumab, an anti-programmed death 1 (PD-1) monoclonal antibody, was assessed as treatment for this condition. METHODS In this randomized, open-label, phase 3 trial, we assigned, in a 2:1 ratio, 361 patients with recurrent squamous-cell carcinoma of the head and neck whose disease had progressed within 6 months after platinum-based chemotherapy to receive nivolumab (at a dose of 3 mg per kilogram of body weight) every 2 weeks or standard, single-agent systemic therapy (methotrexate, docetaxel, or cetuximab). The primary end point was overall survival. Additional end points included progression-free survival, rate of objective response, safety, and patient-reported quality of life. RESULTS The median overall survival was 7.5 months (95% confidence interval [CI], 5.5 to 9.1) in the nivolumab group versus 5.1 months (95% CI, 4.0 to 6.0) in the group that received standard therapy. Overall survival was significantly longer with nivolumab than with standard therapy (hazard ratio for death, 0.70; 97.73% CI, 0.51 to 0.96; P=0.01), and the estimates of the 1-year survival rate were approximately 19 percentage points higher with nivolumab than with standard therapy (36.0% vs. 16.6%). The median progression-free survival was 2.0 months (95% CI, 1.9 to 2.1) with nivolumab versus 2.3 months (95% CI, 1.9 to 3.1) with standard therapy (hazard ratio for disease progression or death, 0.89; 95% CI, 0.70 to 1.13; P=0.32). The rate of progression-free survival at 6 months was 19.7% with nivolumab versus 9.9% with standard therapy. The response rate was 13.3% in the nivolumab group versus 5.8% in the standard-therapy group. Treatment-related adverse events of grade 3 or 4 occurred in 13.1% of the patients in the nivolumab group versus 35.1% of those in the standard-therapy group. Physical, role, and social functioning was stable in the nivolumab group, whereas it was meaningfully worse in the standard-therapy group. CONCLUSIONS Among patients with platinum-refractory, recurrent squamous-cell carcinoma of the head and neck, treatment with nivolumab resulted in longer overall survival than treatment with standard, single-agent therapy. (Funded by Bristol-Myers Squibb; CheckMate 141 ClinicalTrials.gov number, NCT02105636 .). application/pdf https://sonar.ch/global/documents/88264 eng info:eu-repo/semantics/altIdentifier/doi/10.1056/NEJMoa1602252 info:eu-repo/semantics/altIdentifier/pmid/27718784 info:eu-repo/semantics/openAccess The New England journal of medicine. - 2016 Adult Aged Aged, 80 and over Antibodies, Monoclonal Antineoplastic Agents B7-H1 Antigen Carcinoma, Squamous Cell Female Head and Neck Neoplasms Humans Male Neoplasm Recurrence, Local Nivolumab Quality of Life Survival Analysis Nivolumab for Recurrent Squamous-Cell Carcinoma of the Head and Neck. http://purl.org/coar/resource_type/c_6501