Topical bleomycin treatment of oral leukoplakia: A randomized double-blind clinical trial

Background. Oral leukoplakia and oral erythroplakia may be associated with benign and dysplastic cellular changes, and are at risk of malignant transformation. Additional means of management of these lesions is needed. The results of nonblinded trials using topical bleomycin in oral leukoplakia indicated the need for phase III study. Methods. A prospective, double‐blind, randomized trial of topical bleomycin versus placebo was conducted. Bleomycin 1% in dimethylsulphoxide (DMSO) or the carrier was applied for 5 minutes for 14 consecutive days. Clinical assessment and pre‐application and post‐treatment biopsies were conducted. Results. Twenty‐two patients were randomized. Of the patients who received bleomycin, decrease in clinical size of the lesion was achieved (p = 0.001), and histological reduction in dysplasia was seen (p = 0.094). Conclusions. The topical application of bleomycin in DMSO may represent an additional approach to management of oral leukoplakia. The treatment is well‐tolerated, and may be considered when the location or extent of the lesion may make surgical excision difficult. © 1994 John Wiley & Sons, Inc.