Effectiveness and safety profile of ipilimumab therapy in previously treated patients with unresectable or metastatic melanoma - the Romanian Patient Access Program.

Purpose: The Romanian Patient Access Program (Ro-PAP, CA 184-427), part of the European Expanded Access Program (EAP), was developed to evaluate the effectiveness and safety profile ipilimumab in previously treated patients with advanced melanoma (unresectable or metastatic melanoma). The objective of our retrospective observational study of patients included in this program was to provide data recorded in real-life settings.

Methods: We analysed 89 patients enrolled in Ro-PAP, CA 184-427 (54 men and 35 women) aged between 29 and 89 years. The patients received ipilimumab 3mg/kg, administered with short 30-min i.v. infusion every 3 weeks, having a total of 4 doses. Patients were assessed for tumor response, overall survival (OS) and progression-free survival (PFS), and were monitored for adverse events (AE).

Results: At 12 weeks after the completion of therapy, the complete and partial response rates were 6.74% each, stable disease 15.73%, with the best overall response rate 13.48% and disease control rate 29.21%. Median OS was 189.00 days (95% CI 69.50-308.49) and median PFS 124.00 days (95% CI 85.05-162.94). The level of patient functionality at the beginning of ipilimumab treatment showed to be an important predictor of outcome, as patients with ECOG performance status (PS) (0) before therapy with ipilimumab had a higher OS compared with those with impaired functionality.

Conclusions: The use of ipilimumab in daily clinical practice demonstrated to be effective and safe, consistent with data coming from randomized clinical trials or other observational studies.