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J Clin Oncol. 2017 Jan 10;35(2):226-235. Epub 2016 Nov 7.

Efficacy and Safety of Nivolumab Alone or in Combination With Ipilimumab in Patients With Mucosal Melanoma: A Pooled Analysis.

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Sandra P. D'Angelo, Alexander N. Shoushtari, and Jedd D. Wolchok, Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College, New York, NY; James Larkin, Royal Marsden Hospital, London; Paul Lorigan, University of Manchester, Manchester, United Kingdom; Jeffrey A. Sosman, Vanderbilt University Medical Center, Nashville, TN; Celeste Lebbé, Saint-Louis Hospital, Institut National de la Santé et de la Recherche Médicale U976, Université Paris Diderot, Paris; Julie Charles, Grenoble University Hospital, Grenoble Alps University, Grenoble, France; Benjamin Brady, Cabrini Health, Melbourne, Australia; Bart Neyns, Universitair Ziekenhuis Brussel, Brussels, Belgium; Henrik Schmidt, Århus University, Åarhus, Denmark; Jessica C. Hassel, University Hospital Heidelberg, Heidelberg; Peter Mohr, Elbe Kliniken Buxtehude, Buxtehude; Martin Kaatz, SRH Waldklinikum Gera, University Hospital Jena, Jena, Germany; F. Stephen Hodi, Dana-Farber Cancer Institute, Boston, MA; Kerry J. Savage, BC Cancer Agency, University of British Columbia, Vancouver; Wilson H. Miller Jr, Lady Davis Institute and Jewish General Hospital, McGill University, Montreal, Canada; Ivan Marquez-Rodas, Hospital General Universitario Gregorio Marañón, Madrid, Spain; Mario Sznol, Yale University School of Medicine and Smilow Cancer Center, Yale-New Haven Hospital, New Haven, CT; Jeffrey S. Weber, Moffitt Cancer Center, Tampa, FL; and Mary Ruisi and Joel Jiang, Bristol-Myers Squibb, Princeton, NJ.


Purpose Mucosal melanoma is an aggressive malignancy with a poor response to conventional therapies. The efficacy and safety of nivolumab (a programmed death-1 checkpoint inhibitor), alone or combined with ipilimumab (a cytotoxic T-lymphocyte antigen-4 checkpoint inhibitor), have not been reported in this rare melanoma subtype. Patients and Methods Data were pooled from 889 patients who received nivolumab monotherapy in clinical studies, including phase III trials; 86 (10%) had mucosal melanoma and 665 (75%) had cutaneous melanoma. Data were also pooled for patients who received nivolumab combined with ipilimumab (n = 35, mucosal melanoma; n = 326, cutaneous melanoma). Results Among patients who received nivolumab monotherapy, median progression-free survival was 3.0 months (95% CI, 2.2 to 5.4 months) and 6.2 months (95% CI, 5.1 to 7.5 months) for mucosal and cutaneous melanoma, with objective response rates of 23.3% (95% CI, 14.8% to 33.6%) and 40.9% (95% CI, 37.1% to 44.7%), respectively. Median progression-free survival in patients treated with nivolumab combined with ipilimumab was 5.9 months (95% CI, 2.8 months to not reached) and 11.7 months (95% CI, 8.9 to 16.7 months) for mucosal and cutaneous melanoma, with objective response rates of 37.1% (95% CI, 21.5% to 55.1%) and 60.4% (95% CI, 54.9% to 65.8%), respectively. For mucosal and cutaneous melanoma, respectively, the incidence of grade 3 or 4 treatment-related adverse events was 8.1% and 12.5% for nivolumab monotherapy and 40.0% and 54.9% for combination therapy. Conclusion To our knowledge, this is the largest analysis of data for anti-programmed death-1 therapy in mucosal melanoma to date. Nivolumab combined with ipilimumab seemed to have greater efficacy than either agent alone, and although the activity was lower in mucosal melanoma, the safety profile was similar between subtypes.

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