Nivolumab in combination with ipilimumab appears to be more efficacious than either agent alone in cutaneous and mucosal melanoma, with greater activity in cutaneous melanoma than mucosal melanoma, according to a study published in the Journal of Clinical Oncology.1

Mucosal melanoma is an aggressive rare form of cancer that poorly responds to conventional therapies. Although the efficacy and safety of nivolumab monotherapy and combined with ipilimumab have been established for patients with cutaneous melanoma, the impact of nivolumab with or without ipilimumab in mucosal melanoma has not been established.

To assess the efficacy and safety of nivolumab monotherapy and combination therapy with ipilimumab in mucosal melanoma, investigators pooled clinical trial data from 889 patients who received nivolumab monotherapy and 361 who were given nivolumab plus ipilimumab. Of those, 991 had cutaneous melanoma and 121 had mucosal melanoma.

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Median progression-free survival in patients treated with single-agent nivolumab was 3.0 months (95% CI, 2.2-5.4) for mucosal melanoma and 6.2 months (95% CI, 5.1-7.5) for cutaneous melanoma; objective response rates were 23.3% (95% CI, 14.8-33.6) and 40.9% (95% CI, 37.1-44.7), respectively.

Among those who received combination immunotherapy, median progression-free survival was 5.9 months (95% CI, 2.8-not reached) in mucosal melanoma and 11.7 months (95% CI, 8.9-16.7) in cutaneous melanoma, with 37.1% (95% CI, 21.5-55.1) and 60.4% (95% CI, 54.9-65.8) achieving an objective response, respectively.

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Although activity was lower in mucosal melanoma than cutaneous melanoma, the safety profile was similar between the 2 subtypes. The incidence of grade 3 to 4 treatment-related adverse events in mucosal melanoma was 8.1% with nivolumab monotherapy and 40.0% with combination therapy compared with 12.5% and 54.9% in cutaneous melanoma, respectively.

Although these findings suggest that nivolumab with or without ipilimumab is active among patients with this aggressive form of cancer, future large clinical trials are warranted to validate the efficacy and safety of this treatment strategy.      


  1. D’Angelo SP, Larkin J, Sosman JA, et al. Efficacy and safety of nivolumab alone or in combination with ipilimumab in patients with mucosal melanoma: A pooled analysis. J Clin Oncol. 2016 Nov 7. doi: 10.1200/JCO.2016.67.9258. [Epub ahead of print]