Sammendrag
Ipilimumab was the first treatment that improved survival in advanced melanoma.
Efficacy and toxicity in a real-world setting may differ from clinical trials, due to more
liberal eligibility criteria and less intensive monitoring. Moreover, high costs and lack
of biomarkers have raised cost-benefit concerns about ipilimumab in national
healthcare systems and limited its use. Here, we report the prospective, interventional
study, Ipi4 (NCT02068196), which aimed to investigate the toxicity and efficacy
of ipilimumab in a real-world population with advanced melanoma. This
national, multicentre, phase IV trial included 151 patients. Patients received
ipilimumab 3 mg/kg intravenously and were followed for at least 5 years or until
death. Treatment interruption or cessation occurred in 38%, most frequently due to
disease progression (19%). Treatment-associated grade 3 to 4 toxicity was observed
in 28% of patients, and immune-related toxicity in 56%. The overall response rate
was 9%. Median overall survival was 12.1 months (95% CI: 8.3-15.9); and
progression-free survival 2.7 months (95% CI: 2.6-2.8). After 5 years, 20% of patients
were alive. In a landmark analysis from 6 months, improved survival was associated
with objective response (HR 0.16, P = .001) and stable disease (HR 0.49, P = .005)
compared to progressive disease. Poor performance status, elevated lactate dehydrogenase
and C-reactive protein were identified as biomarkers. This prospective trial
represents the longest reported follow-up of a real-world melanoma population
treated with ipilimumab. Results indicate safety and efficacy comparable to phase III
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