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

Journal of Translational Medicine

Paola Queirolo^1,13  Massimo Aglietta¹  Gaetana Rinaldi^1,14  Carolina Cimminiello³  Mario Mandalà⁷  Riccardo Marconcini⁵  Michele Guida⁶  Maria Grazia Bernengo^1,17  Alessandro Testori^1,10  Francesco Cognetti⁹  Ruggero Ridolfi^1,16  Paolo Marchetti²  Lorenza Di Guardo^1,12  Michele Del Vecchio^1,12  Maresa Altomonte⁸  Michele Maio⁸  Jacopo Pigozzo^1,11  Vanna Chiarion Sileni^1,11  Ester Simeone^1,15  Paolo A Ascierto⁴ 

[1] Institute of Cancer Research and Treatment, Piedmont Oncology Foundation, Candiolo, Italy;Sant’ Andrea Hospital, University Sapienza, Rome, Italy;San Raffaele Hospital, Milan, Italy;Unit of Melanoma, Cancer Immunotherapy and Innovative Therapy Unit, Istituto Nazionale Tumori Fondazione ‘G. Pascale’ Via Mariano Semmola, 80131 Napoli, Italy;University Hospital Pisa, ‘Gathered Hospitals of Santa Chiara’, Pisa, Italy;National Cancer Research Center, ‘Giovanni Paolo II’, Bari, Italy;‘Papa Giovanni XXIII’ Hospital, Bergamo, Italy;Istituto Toscano Tumori, University Hospital of Siena, Siena, Italy;Regina Elena National Cancer Institute, Rome, Italy;Divisione Melanoma e Sarcomi Muscolo-Cutanei, Istituto Europeo di Oncologia, Milan, Italy;Veneto Institute of Oncology IOV-IRCCS, Padua, Italy;National Cancer Institute, Milan, Italy;National Institute for Cancer Research, San Martino Hospital, Genoa, Italy;‘Paolo Giaccone’ Polyclinic University Hospital, Palermo, Italy;Istituto Nazionale Tumori Fondazione ‘G. Pascale’, Napoli, Italy;Romagna National Cancer Institute, Meldola, Italy;University Hospital St John the Baptist, Turin, Italy

关键词: Safety;    Melanoma;    Ipilimumab;    Expanded access programme;    Efficacy;   

Others  :  814115
DOI  :  10.1186/1479-5876-12-116

 received in 2014-03-24, accepted in 2014-03-24,  发布年份 2014

【 摘 要 】

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.

【 授权许可】

   
2014 Ascierto et al.; licensee BioMed Central Ltd.

【 图 表 】

Figure 1.

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