Randomized controlled trial of topical mitomycin C for ocular surface squamous neoplasia - Early resolution

Objective: To assess the efficacy and safety of mitomycin C in the treatment of ocular surface squamous neoplasia. Design: Prospective, randomized, controlled, masked, single-center trial. Participants: Forty-eight consecutive patients with biopsy-proven noninvasive ocular surface squamous neoplasia. Intervention: Mitomycin C (0.4 mg/ml) or a placebo was administered as 1 drop 4 times a day for 3 weeks, with crossover of drops if there was no regression within 6 weeks. Main Outcome Measures: Successful treatment was measured as complete resolution of the neoplasia by slit-lamp examination within 6 to 8 weeks of treatment, with a secondary end point of histological resolution by 6 weeks. Results: None of 20 in the placebo group resolved clinically, whereas 24 of 26 in the mitomycin group resolved, giving a relative resolution rate of mitomycin versus placebo of 40.87 (95% confidence interval [CI], 1.01-1656.8). There were no complications. There was no statistical treatment effect on histological resolution, with a resolution rate of mitomycin versus placebo of 1.37 (95% CI, 0.34-5.42). Conclusions: Mitomycin C (0.4 mg/ml) eyedrops used 4 times a day for 3 weeks is an effective agent to cause early clinical resolution of noninvasive ocular surface squamous neoplasia.