RT Journal Article SR Electronic T1 Ipilimumab plus nivolumab for patients with metastatic uveal melanoma: a multicenter, retrospective study JF Journal for ImmunoTherapy of Cancer JO J Immunother Cancer FD BMJ Publishing Group Ltd SP e000331 DO 10.1136/jitc-2019-000331 VO 8 IS 1 A1 Yana G Najjar A1 Kristina Navrazhina A1 Fei Ding A1 Roma Bhatia A1 Katy Tsai A1 Kelly Abbate A1 Barbara Durden A1 Zeynep Eroglu A1 Shailender Bhatia A1 Song Park A1 Akansha Chowdhary A1 Sunandana Chandra A1 Jonathan Kennedy A1 Igor Puzanov A1 Marc Ernstoff A1 Pankit Vachhani A1 Joseph Drabick A1 Arun Singh A1 Tan Xu A1 Jessica Yang A1 Richard Carvajal A1 Daniel Manson A1 John M Kirkwood A1 Justine Cohen A1 Ryan Sullivan A1 Douglas Johnson A1 Pauline Funchain A1 Alexander Shoushtari YR 2020 UL http://jitc.bmj.com/content/8/1/e000331.abstract AB Background Uveal melanoma (UM) is the most common intraocular malignancy in adults. In contrast to cutaneous melanoma (CM), there is no standard therapy, and the efficacy and safety of dual checkpoint blockade with nivolumab and ipilimumab is not well defined.Methods We conducted a retrospective analysis of patients with metastatic UM (mUM) who received treatment with ipilimumab plus nivolumab across 14 academic medical centers. Toxicity was graded using National Cancer Institute Common Terminology Criteria for Adverse Events V.5.0. Progression-free survival (PFS) and overall survival (OS) were calculated using Kaplan-Meier methodology.Results 89 eligible patients were identified. 45% had received prior therapy, which included liver directed therapy (29%), immunotherapy (21%), targeted therapy (10%) and radiation (16%). Patients received a median 3 cycles of ipilimumab plus nivolumab. The median follow-up time was 9.2 months. Overall response rate was 11.6%. One patient achieved complete response (1%), 9 patients had partial response (10%), 21 patients had stable disease (24%) and 55 patients had progressive disease (62%). Median OS from treatment initiation was 15 months and median PFS was 2.7 months. Overall, 82 (92%) of patients discontinued treatment, 34 due to toxicity and 27 due to progressive disease. Common immune-related adverse events were colitis/diarrhea (32%), fatigue (23%), rash (21%) and transaminitis (21%).Conclusions Dual checkpoint inhibition yielded higher response rates than previous reports of single-agent immunotherapy in patients with mUM, but the efficacy is lower than in metastatic CM. The median OS of 15 months suggests that the rate of clinical benefit may be larger than the modest response rate.