Cancer Immunology, Immunotherapy

, Volume 61, Issue 1, pp 41–48 | Cite as

Ipilimumab in pretreated patients with metastatic uveal melanoma: safety and clinical efficacy

  • Riccardo Danielli
  • Ruggero Ridolfi
  • Vanna Chiarion-Sileni
  • Paola Queirolo
  • Alessandro Testori
  • Ruth Plummer
  • Monica Boitano
  • Luana Calabrò
  • Costanza De Rossi
  • Anna Maria Di Giacomo
  • Pier Francesco Ferrucci
  • Laura Ridolfi
  • Maresa Altomonte
  • Clelia Miracco
  • Angelo Balestrazzi
  • Michele Maio
Original article


Current systemic treatments for metastatic uveal melanoma (UM) have not improved overall survival (OS). The fully human anti-cytotoxic T-lymphocyte antigen-4 (CTLA-4) monoclonal antibody, ipilimumab, improved OS of patients with advanced cutaneous melanoma in a phase 3 trial; however, UM patients were excluded. The aim of this subanalysis, performed by the ipilimumab-ocular melanoma expanded access program (I-OMEAP) study group, was to assess the activity and safety of ipilimumab in patients with UM in a setting similar to daily clinical practice. Patients participating in a multicenter expanded access program (EAP) received induction treatment with ipilimumab 10 mg/kg. Maintenance doses were administered in patients who experienced clinical benefit or at physicians’ discretion. Tumor assessment was evaluated per modified World Health Organization criteria at baseline, Week 12, Week 24, and Week 36. Adverse events (AEs) and immune-related AEs (irAEs) were collected according to Common Terminology Criteria for Adverse Events version 3.0. Thirteen pretreated patients with metastatic UM were treated at 6 European institutions. All patients received at least one dose of ipilimumab. Overall, no objective responses were observed; however, two patients had stable disease (SD), with a third patient achieving SD after initial progressive disease. Median OS as of July 1, 2011, was 36 weeks (range 2–172+ weeks). No grade 3/4 AEs of non-immune origin were reported. Three patients (23%) experienced grade 3 irAEs (1 thrombocytopenia, 1 diarrhea, and 1 alanine/aspartate aminotransferase elevation) that resolved with steroid therapy. The results indicate UM is a potential indication for ipilimumab treatment that should be further investigated in clinical trials.


Uveal melanoma Ipilimumab Cytotoxic T-lymphocyte antigen-4 Melanoma Immunotherapy 



The expanded access program was sponsored by Bristol-Myers Squibb. Editorial assistance was provided by StemScientific, funded by Bristol-Myers Squibb. The authors thank Dr Giorgio Frosini and Dr Mario Marini from the Gastroenterology Unit of the University Hospital of Siena for providing the colonoscopic pictures. They also thank the nurses (Angela Iacovelli, Sergio Speranza, Massimo Resti, and Marilena Piccinelli) and data managers (Giovanni Amato and Eliana Pittiglio) at the Medical Oncology and Immunotherapy Unit of the University Hospital of Siena for their contribution to the data collection.


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Copyright information

© Springer-Verlag 2011

Authors and Affiliations

  • Riccardo Danielli
    • 1
  • Ruggero Ridolfi
    • 2
  • Vanna Chiarion-Sileni
    • 3
  • Paola Queirolo
    • 4
  • Alessandro Testori
    • 5
  • Ruth Plummer
    • 6
  • Monica Boitano
    • 4
  • Luana Calabrò
    • 1
  • Costanza De Rossi
    • 3
  • Anna Maria Di Giacomo
    • 1
  • Pier Francesco Ferrucci
    • 5
  • Laura Ridolfi
    • 2
  • Maresa Altomonte
    • 1
  • Clelia Miracco
    • 7
  • Angelo Balestrazzi
    • 8
  • Michele Maio
    • 1
  1. 1.Medical Oncology and Immunotherapy UnitUniversity Hospital of Siena, Istituto Toscano TumoriSienaItaly
  2. 2.Scientific Institute of Romagna for the Study and Treatment of Cancer (IRST)Meldola-ForlìItaly
  3. 3.Istituto Oncologico VenetoPadovaItaly
  4. 4.National Institute for Cancer ResearchGenoaItaly
  5. 5.European Institute of OncologyMilanItaly
  6. 6.Newcastle UniversityNewcastle upon TyneUK
  7. 7.PathologyUniversity Hospital of Siena, Istituto Toscano TumoriSienaItaly
  8. 8.OphthalmologyUniversity Hospital of Siena, Istituto Toscano TumoriSienaItaly

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