RT Journal Article SR Electronic T1 Clinical activity of PD-1 inhibition in the treatment of locally advanced or metastatic basal cell carcinoma JF Journal for ImmunoTherapy of Cancer JO J Immunother Cancer FD BMJ Publishing Group Ltd SP e004839 DO 10.1136/jitc-2022-004839 VO 10 IS 5 A1 In, Gino Kim A1 Nallagangula, Aparna A1 Choi, Jacob Seung A1 Tachiki, Lisa A1 Blackburn, Matthew J A1 Capone, Stephen A1 Bollin, Kathryn B A1 Reuben, Daniel Y. A1 Shirai, Keisuke A1 Zhang-Nunes, Sandy A1 Ragab, Omar A1 Terando, Alicia A1 Hu, Jenny C. A1 Lee, Han A1 Bhatia, Shailender A1 Chandra, Sunandana A1 Lutzky, Jose A1 Gibney, Geoffrey Thomas YR 2022 UL http://jitc.bmj.com/content/10/5/e004839.abstract AB Background Basal cell carcinoma (BCC) is the most common malignancy worldwide, yet the management of patients with advanced or metastatic disease is challenging, with limited treatment options. Recently, programmed death receptor 1 (PD-1) inhibition has demonstrated activity in BCC after prior Hedgehog inhibitor treatment.Methods We conducted a multicenter, retrospective analysis of BCC patients treated with PD-1 inhibitor therapy. We examined the efficacy and safety of PD-1 therapy, as well as clinical and pathological variables in association with outcomes. Progression-free survival (PFS), overall survival (OS) and duration of response (DOR) were calculated using Kaplan-Meier methodology. Toxicity was graded per Common Terminology Criteria for Adverse Events V.5.0.Results A total of 29 patients with BCC who were treated with PD-1 inhibition were included for analysis, including 20 (69.0%) with locally advanced and 9 (31.0%) with metastatic disease. The objective response rate was 31.0%, with five partial responses (17.2%), and four complete responses (13.8%). Nine patients had stable disease (31.0%), with a disease control rate of 62.1%. The median DOR was not reached. Median PFS was 12.2 months (95% CI 0.0 to 27.4). Median OS was 32.4 months (95% CI 18.1 to 46.7). Two patients (6.9%) developed grade 3 or higher toxicity, while four patients (13.8%) discontinued PD-1 inhibition because of toxicity. Higher platelets (p=0.022) and any grade toxicity (p=0.024) were significantly associated with disease control rate.Conclusions The clinical efficacy of PD-1 inhibition among patients with advanced or metastatic BCC in this real-world cohort were comparable to published trial data. Further investigation of PD-1 inhibition is needed to define its optimal role for patients with this disease.Data are available on reasonable request.