Clinical activity of PD-1 inhibition in the treatment of locally advanced or metastatic basal cell carcinoma

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 inhibit...

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Published in:Journal for immunotherapy of cancer 2022-05, Vol.10 (5), p.e004839
Main Authors: In, Gino Kim, Nallagangula, Aparna, Choi, Jacob Seung, Tachiki, Lisa, Blackburn, Matthew J, Capone, Stephen, Bollin, Kathryn B, Reuben, Daniel Y, Shirai, Keisuke, Zhang-Nunes, Sandy, Ragab, Omar, Terando, Alicia, Hu, Jenny C, Lee, Han, Bhatia, Shailender, Chandra, Sunandana, Lutzky, Jose, Gibney, Geoffrey Thomas
Format: Article
Language:English
Subjects:
Carcinoma, Basal Cell - drug therapy
Carcinoma, Basal Cell - pathology
Clinical/Translational Cancer Immunotherapy
Hedgehog Proteins
Humans
Programmed Cell Death 1 Receptor - therapeutic use
Retrospective Studies
Skin Neoplasms - drug therapy
Skin Neoplasms - pathology
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Summary: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. 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. 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. 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.
ISSN:2051-1426
2051-1426
DOI:10.1136/jitc-2022-004839