Retrospective multicenter analysis of real-life toxicity and outcome of ipilimumab and nivolumab in metastatic uveal melanoma.

Publication date: Jun 24, 2025

Uveal melanoma (UM) is the most common primary ocular malignancy with a high rate of metastases. While immune checkpoint inhibitors (ICIs), including ipilimumab and nivolumab (ipi+nivo), have shown efficacy in metastatic cutaneous melanoma, their success in metastatic UM (MUM) remains limited. This study evaluates toxicity and outcomes of ipi+nivo in the largest, multicenter MUM cohort. We analyzed 131 MUM patients treated with ipi+nivo from 2016-2024 across five international centers. Rates of toxicity, response, and survival outcomes were assessed. Among 131 patients, 37. 4% of patients received four cycles of ipi+nivo. The most common reason for ipi+nivo discontinuation (31. 3%) was toxicity. Of all treated patients, 80. 2% experienced immune-related adverse events (irAEs). The overall response rate (ORR) was 16. 4%, and the disease control rate (DCR) was 43. 4%. Progression-free survival (PFS) was three months, and the median overall survival (OS) was 18 months. Patients receiving ipi+nivo as second-line therapy had lower ORR compared to patients who received ipi+nivo as first-line therapy (p = 0. 04). Patients with exclusively extrahepatic metastases had a better ORR and OS compared to those with hepatic or mixed metastases (p = 0. 02, p = 0. 02, respectively). 20. 6% of patients developed eosinophilia during treatment, which was associated with improved median OS (24 months vs. 15 months, p = 0. 02). Ipi+nivo shows moderate efficacy and clinically relevant toxicities in patients with MUM. Eosinophilia is a potential prognostic biomarker, that merits further investigation.

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