{"doi":"10.1136/jitc-2022-004839","title":"Clinical activity of PD-1 inhibition in the treatment of locally advanced or metastatic basal cell carcinoma","abstract":"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.","journal":"Journal for ImmunoTherapy of Cancer","year":2022,"id":269601,"datarank":0.41588830833596724,"base_score":2.772588722239781,"endowment":2.772588722239781,"self_citation_contribution":0.41588830833596724,"citation_network_contribution":0.0,"self_endowment_contribution":0.41588830833596724,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":15,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.8907,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2022-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":933302,"name":"Aparna Nallagangula","orcid":null,"position":1,"is_corresponding":false},{"id":653426,"name":"Jacob Choi","orcid":"0000-0002-5496-6033","position":2,"is_corresponding":false},{"id":820717,"name":"Lisa Tachiki","orcid":"0000-0001-8057-8029","position":3,"is_corresponding":false},{"id":933303,"name":"Matthew Blackburn","orcid":null,"position":4,"is_corresponding":false},{"id":6584,"name":"Stephen Capone","orcid":"0000-0001-7231-1535","position":5,"is_corresponding":false},{"id":320441,"name":"Kathryn Bollin","orcid":"0000-0001-7343-8925","position":6,"is_corresponding":false},{"id":618582,"name":"Daniel Y. 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Terando","orcid":"0000-0002-6319-2463","position":11,"is_corresponding":false},{"id":798793,"name":"Jenny Hu","orcid":null,"position":12,"is_corresponding":false},{"id":785777,"name":"Han Lee","orcid":"0000-0002-6633-966X","position":13,"is_corresponding":false},{"id":230463,"name":"Shailender Bhatia","orcid":"0000-0002-3816-2238","position":14,"is_corresponding":false},{"id":268510,"name":"Sunandana Chandra","orcid":"0000-0001-7936-5069","position":15,"is_corresponding":false},{"id":290806,"name":"Jose Lutzky","orcid":"0000-0002-9503-2130","position":16,"is_corresponding":false},{"id":530719,"name":"Geoffrey T. Gibney","orcid":"0000-0002-5401-8498","position":17,"is_corresponding":false},{"id":748460,"name":"Gino K. 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