Background: Patients with advanced uveal melanoma have a poor prognosis and limited treatment options. Ipilimumab is approved for pre-treated adult patients with advanced melanoma. However, because previous clinical trials with ipilimumab have excluded patients with uveal melanoma, data in this patient population are limited. Patients and methods: Pre-treated patients with advanced uveal melanoma received ipilimumab 3 mg/kg through an expanded access programme, every 3 weeks for four doses. Tumour assessments were conducted at baseline and after completion of treatment and patients were monitored throughout for adverse events. Results: Among 82 assessable patients, 4 (5%) had an immune-related objective response and 24 (29%) had immunerelated stable disease lasting ≥3 months for an immune-related disease control rate of 34%. With a median follow-up of 5.6 months, median overall survival (OS) was 6.0 months and median progression-free survival (PFS) was 3.6 months. The 1-year rates of OS and PFS were 31% and 11%, respectively. The safety profile of ipilimumab was similar to that in patients with cutaneous melanoma. Conclusions: These data suggest ipilimumab 3 mg/kg is a feasible option in pre-treated patients with metastatic uveal melanoma. Evidence of disease control and a 1-year survival rate of 31% indicate the need for further investigation in randomised, controlled trials to determine the optimal timing and use of ipilimumab in this patient population.

Original languageEnglish
Article numbermdt376
Pages (from-to)2911-2915
Number of pages5
JournalAnnals of Oncology
Issue number11
Publication statusPublished - Nov 2013


  • Efficacy
  • Expanded access programme
  • Ipilimumab
  • Metastatic melanoma
  • Safety
  • Uveal melanoma

ASJC Scopus subject areas

  • Oncology
  • Hematology


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