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

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.

CINAHL, Cochrane Library, PubMed, and SCOPUS.

Data sources 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.

A total of 9 studies reporting on (N = 89) patients with (n = 99) lesions were included. The average age was 48.4 (range 43-66), and 48.6% were male. 5% topical imiquimod was used in varying treatment regimens, most commonly for 6 weeks in duration (7/9 papers). 34.3% of lesions had complete resolution, 43.4% had incomplete resolution, and 22.2% had no change in the lesion. The most commonly reported adverse effects were burning/soreness at the site (73.0%), fatigue (22.5%), oral lichen planus development or flare (11.2%), and flu-like symptoms (2.2%).

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.