Topical Imiquimod for Oral Cavity Leukoplakia and Dysplasia: A Scoping Review

Introduction: Imiquimod, a toll-like receptor 7 agonist, is currently FDA-approved for treatment of actinic keratoses, superficial BCC, and external genital warts. It has also demonstrated promise in off-label use in the treatment of oral cavity leukoplakia and dysplasia. The aim of this scoping review is to summarize the current literature on this novel treatment strategy.

Methods: CINAHL, Cochrane Library, PubMed, and SCOPUS databases were searched from inception through December 2024. The search was performed in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analysis Extension for Scoping Reviews. Studies reporting on oral leukoplakia or dysplasia treated with topical Imiquimod were included, with primary outcome being lesion resolution or reduction.

Results: A total of 9 studies reporting on (N = 89) patients with (n = 99) lesions were included. The average age was 48.36 (range 43-66), and 51.43% were male. 5% topical Imiquimod was used in varying treatment regimens, most commonly for 6 weeks in duration (7/9 papers). 33.33% of lesions had complete resolution, 33.33% had incomplete resolution, and 22.22% had no change in the lesion. The most commonly reported adverse effects were burning/soreness at the site (73.03%), fatigue (22.47%), oral lichen planus development or flare (11.24%), and flu-like symptoms (2.25%).

Conclusions: Topical Imiquimod for the treatment of oral cavity leukoplakia and dysplasia is a promising new treatment strategy. Efficacy has been demonstrated in reducing lesion size and even complete lesion resolution, with minimal adverse effects. Further research is needed on this innovative therapeutic approach.