Background:
AM is a rare melanoma subtype with poor prognosis. While retrospective data suggests low activity of PD1 alone, no data is available on the efficacy of combination PD1/CTLA4 in AM. AM primary site is associated with differences in tumour mutation burden, which may impact CPI activity. We examined the efficacy of CPIs in AM, and in primary site subgroups.
Methods:
Patients with unresectable stage III/IV AM treated with ≥1 line of CPI (PD1 and/or ipilimumab (Ipi)) were studied. Disease/patient characteristics and therapy were examined. Multivariable logistic and Cox regression analysis were conducted. Primary outcomes were objective response rate (ORR), progression free survival (PFS) and overall survival (OS).
Results:
369 patients were included; median age at first diagnosis was 63y, 80% Caucasian, 53% male, 12% BRAFm. Median time from primary diagnosis to advanced disease was 19.6 months. Primary site was 260 (70%) plantar, 25 (7%) palmar, 84 (23%) subungual. Excluding 41 patients who received adjuvant CPI, 1st line systemic therapy included 151 (46%) PD1, 51 (15%) Ipi, 54 (16%) combination CPI and 72 (22%) other therapies. At 1st line therapy commencement, 30% were stage M1c, 53% ECOG 0 and 19% elevated LDH. Median follow-up was 8.1y.
ORR was 44%, 26% and 12% for combination CPI, PD1 and CTLA4, respectively (p=0.0015, multivariate analysis). PFS was significantly associated with 1st line therapy, however PFS was not significantly different between PD1 and combination CPI (p=0.42). Median OS was 2.3y (95% CI 1.9-2.6) and did not vary by 1st line therapy (p=0.57). No outcome differences were found between primary site.
Conclusions:
To our knowledge, this is the largest study on CPIs in AM. CPIs are active in advanced AM, with combination CPIs superior to PD1 alone in terms of ORR but not PFS or OS. Primary AM site did not impact CPI efficacy.