Efficacy and safety of ipilimumab monotherapy in patients with pretreated advanced melanoma: a multicenter single-arm phase II study

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Background: This phase II study evaluated the safety and activity of ipilimumab, a fully human mAb that blocks cytotoxic T-lymphocyte antigen-4, in patients with advanced melanoma. Patients and methods: Patients with previously treated, unresectable stage III/stage IV melanoma received 10 mg/kg ipilimumab every 3 weeks for four cycles (induction) followed by maintenance therapy every 3 months. The primary end point was best overall response rate (BORR) using modified World Health Organization (WHO) criteria. We also carried out an exploratory analysis of proposed immune-related response criteria (irRC). Results: BORR was 5.8% with a disease control rate (DCR) of 27% (N = 155). One-and 2-year survival rates (95% confidence interval) were 47.2% (39.5% to 55.1%) and 32.8% (25.4% to 40.5%), respectively, with a median overall survival of 10.2 months (7.6-16.3). Of 43 patients with disease progression by modified WHO criteria, 12 had disease control by irRC (8% of all treated patients), resulting in a total DCR of 35%. Adverse events (AEs) were largely immune related, occurring mainly in the skin and gastrointestinal tract, with 19% grade 3 and 3.2% grade 4. Immune-related AEs were manageable and generally reversible with corticosteroids. Conclusion: Ipilimumab demonstrated clinical activity with encouraging long-term survival in a previously treated advanced melanoma population.


  • S. J. O'Day
  • M. Maio
  • V. Chiarion-Sileni
  • T. F. Gajewski
  • H. Pehamberger
  • I. N. Bondarenko
  • P. Queirolo
  • Lotta Lundgren
  • S. Mikhailov
  • L. Roman
  • C. Verschraegen
  • R. Humphrey
  • R. Ibrahim
  • V. de Pril
  • A. Hoos
  • J. D. Wolchok
Research areas and keywords

Subject classification (UKÄ) – MANDATORY

  • Cancer and Oncology


  • phase II clinical trial, metastatic, melanoma, ipilimumab, cytotoxic T-lymphocyte antigen-4, immunotherapy
Original languageEnglish
Pages (from-to)1712-1717
JournalAnnals of Oncology
Issue number8
Publication statusPublished - 2010
Publication categoryResearch