Aims: Ipilimumab is a fully human monoclonal antibody that blocks cytotoxic T-lymphocyte antigen-4 to augment antitumor immune responses. We report a phase III trial that evaluated Ipilimumab as an adjuvant therapy for resected stage III melanoma at high risk of recurrence.
Methods: In this randomised, double-blind trial, eligible patients included those ≥18 years of age who underwent complete resection of stage III cutaneous melanoma (excluding lymph node metastasis ≤1 mm or in-transit metastasis). 951 patients were randomised (stratified by stage and region) to receive Ipilimumab 10 mg/kg (n=475) or placebo (n=476) every 3 weeks for 4 doses, then every 3 months for up to 3 years until completion, disease recurrence, or unacceptable toxicity. Primary endpoint was recurrence-free survival (RFS), analysed on the intent-to-treat population. 512 RFS events (recurrence or death) were needed to provide 90% power to detect an Ipilimumab vs Placebo hazard ratio (HR) of 0.75 (2-sided α=5%).
Results: Overall, 20%/44%/36% of patients had stage IIIA/IIIB/IIIC. At a median follow-up of 2.7 yrs, Ipilimumab (234 events) significantly improved RFS vs Placebo (294 events): median RFS was 26.1 vs 17.1 months, HR was 0.75 (95% CI; 0.64, 0.90), P=0.0013. RFS benefit was consistent across subgroups. Most common grade 3/4 immune-related adverse events (irAEs) in Ipilimumab and Placebo were gastrointestinal (15.9% vs 0.8%), hepatic (10.6% vs 0.2%), and endocrine (8.5% vs 0%). Most irAEs were managed and resolved using established algorithms. Of 471 patients on Ipilimumab, 52% discontinued due to AEs [182 (38.6%) within 12 weeks]; 5 (1.1%) died due to drug-related AEs.
Conclusions: In this phase III trial, Ipilimumab as adjuvant therapy provided a clinically and statistically significant improvement in RFS vs placebo for patients with stage III melanoma at high risk of recurrence. AE profile was generally consistent with that observed in advanced melanoma, with a higher incidence of endocrinopathies.