The European Ipilimumab Expanded Access Programme (EAP): Efficacy and Safety Data from the Italian Cohort of Patients with Pretreated, Advanced Melanoma

ABSTRACT Purpose Ipilimumab was the first agent approved for the treatment of unresectable or metastatic melanoma that showed an overall survival benefit in a randomised phase III trial. Here, we evaluate the safety and efficacy of ipilimumab treatment outside of clinical trials in patients enrolled in the EAP in Italy. Methods Ipilimumab was available upon physician request for patients aged ≥16 years with unresectable stage III/stage IV melanoma who had either failed systemic therapy or were intolerant to ≥1 systemic treatment and for whom no other therapeutic option was available. Ipilimumab 3 mg/kg was administered intravenously 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 ipilimumab dose using Common Terminology Criteria for Adverse Events v.3.0. Results In total, 848 Italian patients participated in the EAP from June 2010 to April 2012 across 53 centres. Of these 848 patients, data are currently available for 563 patients. With a median follow-up of 3 months, the disease control rate among 468 evaluable patients was 31.4%, including 7 patients with a complete response, 51 with a partial response and 89 with stable disease. As of April 2012, median progression-free survival and overall survival were 3.1 months and 6.2 months, respectively, with 1-year survival rate of 34%. In total, 50.1% patients reported an AE of any grade, most of which were drug-related (36.2%). Grade 3/4 AEs were reported by 18.5% patients and considered drug-related in 8.5%. Eleven patients discontinued treatment due to toxicity. AEs were generally reversible with treatment as per protocol-specific guidelines. Complete data on all 848 patients, with longer follow-up, will be presented. Conclusions Ipilimumab is a feasible treatment option for pretreated patients who progressed on, or were unable to tolerate previous therapies. Many patients experienced durable disease control. Disclosure P.A. Ascierto: PA has served as a consultant for Merck Sharp u0026 Dohme, and as an advisor to Bristol-Myers Squibb (BMS), Merck Sharp u0026 Dohme, Roche, GlaxoSmithKline, Amgen, Celgene, Medimmune and Novartis. He has received honoraria from BMS, Merck Sharp u0026 Dohme and Roche. V. Chiarion Sileni: Vanna Chiaron Sileni has acted as an advisor for Bristol-Myers Squibb, Roche, GlaxoSmithKline, Merck Sharp u0026 Dohme and Schering-Plough. M.G. Bernengo: Maria Grazia Bernengo has acted as an advisor to Bristol-Myers Squibb, Novartis and Pfizer. 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. All other authors have declared no conflicts of interest.