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. 2014 May 7:12:116.
doi: 10.1186/1479-5876-12-116.

Clinical experience with ipilimumab 3 mg/kg: real-world efficacy and safety data from an expanded access programme cohort

Paolo A Ascierto  1 , Ester Simeone, Vanna Chiarion Sileni, Jacopo Pigozzo, Michele Maio, Maresa Altomonte, Michele Del Vecchio, Lorenza Di Guardo, Paolo Marchetti, Ruggero Ridolfi, Francesco Cognetti, Alessandro Testori, Maria Grazia Bernengo, Michele Guida, Riccardo Marconcini, Mario Mandalà, Carolina Cimminiello, Gaetana Rinaldi, Massimo Aglietta, Paola Queirolo
Affiliations

Clinical experience with ipilimumab 3 mg/kg: real-world efficacy and safety data from an expanded access programme cohort

Paolo A Ascierto et al. J Transl Med. .

Abstract

Background: Ipilimumab improves survival in patients with advanced melanoma. The activity and safety of ipilimumab outside of a clinical trial was assessed in an expanded access programme (EAP).

Methods: Ipilimumab was available upon physician request for patients aged 16 or over with pretreated stage III (unresectable)/IV melanoma, for whom no other therapeutic option was available. Patients received ipilimumab 3 mg/kg every 3 weeks for four doses. Patients with stable disease or an objective response to ipilimumab were eligible for retreatment upon disease progression. Tumour assessments were conducted at baseline and week 12. Patients were monitored for adverse events (AEs) within 3 to 4 days of each scheduled visit.

Results: Of 855 patients participating in the EAP in Italy, 833 were evaluable for response. Of these, 13% had an objective immune response, and the immune-related disease control rate was 34%. Median progression-free survival and overall survival were 3.7 and 7.2 months, respectively. Efficacy was independent of BRAF and NRAS mutational status. Overall, 33% of patients reported an immune-related AE (irAE). The frequency of irAEs was not associated with response to ipilimumab.

Conclusions: Outside of a clinical trial setting, ipilimumab is a feasible treatment option in patients with pretreated metastatic melanoma, regardless of BRAF and NRAS mutational status. Data from this large cohort of patients support clinical trial evidence that ipilimumab can induce durable disease control and long-term survival in patients who have failed to respond to prior treatment.

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Figures

Figure 1
Figure 1
Kaplan–Meier curves for PFS and OS. PFS for all patients (A). OS for all patients (B). OS for BRAF-mutation positive (n = 173) and BRAF wildtype (n = 296) patients (C). OS for NRAS-mutation positive (n = 14) and NRAS wildtype (n = 68) patients (D). OS in patients with or without an irAE (any grade) in patients with who received three or four cycles of ipilimumab (E). PFS: progression-free survival; OS: overall survival; irAE: immune-related adverse event.
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