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.

This prospective German NICO study (NCT02990611) evaluated real-world outcomes with nivolumab plus ipilimumab versus nivolumab monotherapy in 755 patients with advanced melanoma, including those with melanoma brain metastases (MBM).

• Treatment groups: 486 received nivolumab plus ipilimumab and 269 received nivolumab alone. Combination-treated patients were younger and had poorer baseline prognostic features. Median follow-up was 46.8 and 38.7 months, respectively.
• MBM population: 221 patients (29.3%) had MBM at baseline; 15 had symptomatic MBM (defined by dexamethasone use).

First-line outcomes by MBM status:

• Nivolumab plus ipilimumab
  • ORR: 46.2% (with MBM) vs 54.0% (without MBM)
  • 3-year OS: 34.0% (with MBM) vs 47.0% (without MBM)
• Nivolumab alone
  • ORR: 61.5% (with MBM) vs 55.1% (without MBM)
  • 3-year OS: 42.7% (with MBM) vs 47.8% (without MBM)

Landmark analysis at 3 months in MBM:

• Among patients achieving CR/PR, 3-year OS was 71.9% with nivolumab plus ipilimumab and 89.6% with nivolumab alone.

MBM symptom status:

• 3-year OS was 42.2% in asymptomatic vs 20.0% in symptomatic MBM.

Safety and QoL:

• Rates of serious grade 3/4 treatment-related adverse events were similar in patients with and without MBM.
• Health-related quality of life remained stable.

Overall, in routine practice, both nivolumab plus ipilimumab and nivolumab monotherapy provided meaningful, durable benefit for a substantial subset of patients with advanced melanoma, including those with asymptomatic brain metastases.