Efficacy and Safety of Ipilimumab in Patients with Pretreated, Mucosal Melanoma: Experience from Italian Clinics Participating in the European Expanded Access Programme (EAP)

ABSTRACT Purpose Ocular melanoma is a rare malignancy with an incidence of 5.3–10.9 cases per million per year (Papastefanou and Cohen; J Skin Cancer 2011). Currently, the treatment of metastatic ocular melanoma is limited by the lack of an effective systemic therapy. The EAP provided an opportunity to assess the activity and safety of ipilimumab in patients with ocular melanoma outside of a controlled clinical trial in patients from the EAP in Italy. Methods Ipilimumab was available upon physician request for patients aged ≥16 years with stage III (unresectable) or stage IV skin, ocular or mucosal melanoma, who had failed or did not tolerate previous treatments and for whom no therapeutic option was available. Patients were treated with ipilimumab 3 mg/kg every 3 weeks for 4 doses. Tumour assessments were conducted at baseline and after completion of induction therapy using immune-related response criteria. Patients were monitored for adverse events (AEs), including immune-related AEs, within 3 to 4 days of each scheduled visit using Common Terminology Criteria for Adverse Events v.3.0. Results Of 848 Italian patients participating in the EAP, 83 (9.8%) had ocular melanoma. Of these 83 patients, 55 have data available. With a median follow-up of 3 months, the disease control rate among 46 evaluable patients was 34.8%, including 3 patients with a partial response and 13 with stable disease. As of April 2012, median progression-free survival and overall survival among patients with ocular melanoma were 2.9 months and 5.9 months, respectively. In total, 63.6% patients reported an AE of any grade, most of which were drug-related (47.3%). Grade 3/4 AEs, which were reported by 20.0% patients, were only considered drug-related in 5.4%. AEs were generally manageable and most resolved with treatment as per protocol-specific guidelines. Conclusions Safety and early efficacy results are similar to experience in other melanoma populations, thus suggesting that treatment of ocular melanoma with ipilimumab warrants further investigation in prospective clinical trials. Disclosure M. Maio: Michele Maio has acted as an advisor for Bristol-Myers Squibb and Roche. V. Chairion Sileni: Vanna Chiaron Sileni has acted as an advisor for Bristol-Myers Squibb, Roche, GlaxoSmithKline, Merck Sharp u0026 Dohme and Schering-Plough. P. Queirolo: Paola Queirolo has acted as an advisor for Roche, GlaxoSmithKline, Bristol-Myers Squibb and Schering-Plough and received honoraria from Bristol-Myers Squibb and Roche. P.A. Ascierto: PA has served as a consultant/advisor for Merck Sharp u0026 Dohme, and as an advisor to Bristol-Myers Squibb (BMS), Roche, GlaxoSmithKline, Amgen, Celgene, Medimmune and Novartis. He has received honoraria from BMS, Merck Sharp u0026 Dohme and Roche. All other authors have declared no conflicts of interest.