RT Journal Article SR Electronic T1 Efficacy of immune checkpoint inhibitors for in-transit melanoma JF Journal for ImmunoTherapy of Cancer JO J Immunother Cancer FD BMJ Publishing Group Ltd SP e000440 DO 10.1136/jitc-2019-000440 VO 8 IS 1 A1 Nan Tie, Emilia A1 Lai-Kwon, Julia A1 Rtshiladze, Michael Alexander A1 Na, Lumine A1 Bozzi, James A1 Read, Tavis A1 Atkinson, Victoria A1 Au-Yeung, George A1 Long, Georgina V A1 McArthur, Grant A A1 Sandhu, Shahneen A1 Saw, Robyn A1 Walpole, Euan A1 Menzies, Alexander A1 Smithers, Mark A1 Gyorki, David E YR 2020 UL http://jitc.bmj.com/content/8/1/e000440.abstract AB Background The efficacy of immune checkpoint inhibitors (ICI) in metastatic melanoma is well established. However, there are limited data regarding their efficacy in in-transit melanoma (ITM). This study assessed the efficacy of ICI in patients with ITM.Methods A retrospective review of patients with ITM commenced on an ICI between March 2013 and February 2018 at three tertiary centers in Australia. Patients were excluded if they had previous or synchronous distant metastases. Overall response rate (ORR), progression-free survival (PFS) and overall survival (OS) were based on a composite of radiological and clinical assessments.Results Fifty-four patients were included: 27 (50%) female; median age 75 (range 26–94); 12 (22%) stage IIIB, 40 (74%) stage IIIC and 2 (4%) stage IIID; 10 (19%) BRAF mutant. Forty (74%) received single-agent anti-PD-1 (pembrolizumab or nivolumab), 8 (15%) single agent anti-CTLA-4 (ipilimumab), 5 (9%) combination anti-PD-1/anti-CTLA-4 (ipilimumab and nivolumab or pembrolizumab) and 1 (2%) combination anti-PD-L1 (atezolizumab) and MEK inhibitor (cobimetinib). The median follow-up was 15 months (2–46).ORR to ICI was 54%: 14 (26%) complete responses; 15 (28%) partial responses; 9 (17%) stable disease; 16 (30%) progressive disease. Thirteen (46%) responders had only one ITM lesion. ORR was 58% for single-agent anti-PD-1, 38% for single-agent anti-CTLA4 and 40% for anti-PD-1/anti-CTLA-4. The median PFS was 11.7 months (6.6-not reached). 1-year and 2-year PFS were 48% and 39%, respectively,. Fourteen progressed locoregionally and 11 progressed distantly. The median OS was not reached. 1-year and 2-year OS were 85% and 63%, respectively. No clinicopathological features were associated with ORR.Conclusions and relevance ICI produce objective responses in ITM and should be considered in patients with unresectable ITM or disease recurrence.