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.

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.