Vitiligo is occasionally seen in
melanoma patients. Although several studies indicate a correlation between
vitiligo occurrence and clinical response in
melanoma patients receiving
immunotherapy, most studies have included heterogeneous patient and treatment settings. The aim of this study is to investigate the correlation between the occurrence of
vitiligo and clinical benefit of
nivolumab treatment in advanced
melanoma patients. We retrospectively reviewed unresectable stage III or IV
melanoma patients treated with
nivolumab. Of 35
melanoma patients treated with
nivolumab, 25.7% (9/35) developed
vitiligo during treatment. The time from the start of
nivolumab treatment to occurrence of
vitiligo ranged 2-9 months (mean, 5.2). Of nine patients who developed
vitiligo, two (22.2%) had a complete response to
nivolumab and two (22.2%) had a partial response. The objective response rate was significantly higher in patients with
vitiligo than in patients without
vitiligo (4/9 [44.4%] vs 2/26 [7.7%]; P = 0.027). The mean time to
vitiligo occurrence in patients achieving an objective response was significantly less than that in patients who showed no response (3.1 vs 6.8 months, P = 0.004).
Vitiligo occurrence was significantly associated with prolonged progression-free and overall survival (hazard ratio, 0.24 and 0.16; 95% confidence interval, 0.11-0.55 and 0.03-0.79; P = 0.005, and 0.047, respectively). At the 20-week landmark analysis, however,
vitiligo was not associated with a statistically significant overall survival benefit (P = 0.28). The occurrence of
vitiligo during
nivolumab treatment may be correlated with favorable clinical outcome.