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

Abstract

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.