- 作者：Michele Del Vecchio ; Lorenza Di Guardo ; Paolo A. Ascierto ; Antonio M. Grimaldi ; Vanna Chiarion Sileni ; Jacopo Pigozzo ; Virginia Ferraresi ; Carmen Nuzzo ; Gaetana Rinaldi ; Aless ; ro Testori ; Pier F. Ferrucci ; Paolo Marchetti ; Federica De Galitiis ; Paola Queirolo ; Elena Tornari ; Riccardo Marconcini ; Luana Calabr貌 ; Michele Maio
- 关键词：Efficacy ; Expanded access programme ; Ipilimumab ; Metastatic melanoma ; Mucosal ; Safety
- 刊名：European Journal of Cancer
- 出版年：January, 2014
- 年：2014
- 卷：50
- 期：1
- 页码：121-127
- 全文大小：536 K
Background
Mucosal melanoma is an extremely rare and aggressive malignancy that often remains undetected until it reaches an advanced stage, when effective treatment options are limited. The activity and safety of ipilimumab were assessed in an Expanded Access Programme (EAP) that included patients with metastatic, mucosal melanoma.Methods
Ipilimumab was available upon physician request for patients aged 猢?6 years with stage III (unresectable) or IV skin, ocular or mucosal melanoma, who had failed or did not tolerate previous treatments and had no other therapeutic option 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 using immune-related response criteria. Patients were monitored for adverse events (AEs), including immune-related AEs, within 3 to 4 days of each scheduled visit.
Results
Of 855 patients participating in the EAP in Italy, 71 (8%) had metastatic, mucosal melanoma. With a median follow-up of 21.8 months, the response rate was 12% and the immune-related disease control rate was 36%. Median progression-free survival and overall survival were 4.3 and 6.4 months, respectively. In total, 34% of patients reported treatment-related AEs of any grade, which were grade 3 or 4 in 9% of patients. AEs were generally manageable as per protocol-specific guidelines.
Conclusion/interpretation
Ipilimumab may be a feasible treatment option in pretreated patients with metastatic mucosal melanoma, and warrants further investigation in prospective clinical trials.