Real‐world effectiveness and safety with nivolumab plus ipilimumab or nivolumab alone in patients with or without melanoma brain metastasis: Results from the German noninterventional NICO study

Abstract The prospective, German NICO study ( ClinicalTrials.gov identifier: NCT02990611) evaluated real‐world effectiveness and safety with nivolumab plus ipilimumab or nivolumab alone (any‐line) in patients with advanced melanoma with/without melanoma brain metastasis (MBM). A total of 755 patients treated with nivolumab plus ipilimumab ( n = 486; median follow‐up, 46.8 months) or nivolumab alone ( n = 269; median follow‐up, 38.7 months) were enrolled. Baseline characteristics differed between the treatment groups, with the nivolumab plus ipilimumab group being younger and having poorer prognostic factors. At baseline, 221 patients (29.3%) had MBM, among whom 15 patients had symptomatic MBM based on dexamethasone use. In patients with/without MBM receiving first‐line nivolumab plus ipilimumab, objective response rates (ORRs) were 46.2% and 54.0%, respectively; 3‐year overall survival (OS) rates were 34.0% and 47.0%. In patients with/without MBM receiving first‐line nivolumab alone, ORRs were 61.5% and 55.1%, respectively; 3‐year OS rates were 42.7% and 47.8%. In a 3‐month landmark analysis, patients with MBM with a complete/partial response demonstrated 3‐year OS rates of 71.9% with nivolumab plus ipilimumab and 89.6% with nivolumab alone. Three‐year OS rates were 42.2% and 20.0% with asymptomatic and symptomatic MBM, respectively. There were no substantial differences in the rates of serious grade 3/4 treatment‐related adverse events between patients with/without MBM. HRQoL was stable. Results from this real‐world study show that a substantial proportion of patients with MBM derive long‐term benefit from nivolumab plus ipilimumab or nivolumab alone, particularly those with asymptomatic MBM.