10-years of first-line ipilimumab and nivolumab in the management of melanoma brain metastases: a single centre experience

Background.To evaluate the efficacy of first-line ipilimumab and nivolumab, alongside local therapies, in patients with melanoma brain metastases (MBM).Methods.This retrospective study included MBM patients treated with first-line ipilimumab and nivolumab in the metastatic setting at the Royal Marsden Hospital, London, between 2013 and 2024.Patient outcomes and associations of local therapies with survival were assessed.Results.A total of 93 MBM patients were identified, including 47 symptomatic patients and 43 patients receiving corticosteroids at treatment initiation.Median follow-up was 55.6 months (95% confidence interval (CI) 36.6-82.1).Median overall survival (OS) for the entire cohort was 9.8 months (95% CI 5.8-29.5),and median progression-free survival (PFS) was 3.4 months (95% CI 2.6-5.9).The intracranial objective response rate (ORR) was 18%.Among symptomatic patients, median OS was 5.9 months (95% CI 5.03-46), median PFS 3.2 months (95% CI 2.1-5.99), and intracranial ORR 13% versus median OS 16.3 months (95% CI 7.85-81.1),median PFS 4.4 months (95% CI 2.63-8.18),and intracranial ORR 24% in asymptomatic patients.Eastern Cooperative Oncology Group performance status 2 and elevated lactate dehydrogenase were independently associated with worse outcomes in multivariable analysis.Local therapy alongside systemic treatment was associated with a non-significant trend toward improved survival (median OS 18.3 months 95% CI 11.6-NR vs. 5.4 months 95% CI 4.3-29.5,HR 0.68 0.4-1.13).Conclusions.This retrospective study supports the real-world efficacy of first-line ipilimumab and nivolumab in asymptomatic MBM.Outcomes in symptomatic or corticosteroid-dependent patients remained poor.