Randomized controlled trial of topical mitomycin C for ocular surface squamous neoplasia: early resolution

Ophthalmology. 2007 May;114(5):976-82. doi: 10.1016/j.ophtha.2006.09.026. Epub 2007 Jan 22.


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.

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Topical
  • Adult
  • Aged
  • Aged, 80 and over
  • Antibiotics, Antineoplastic / administration & dosage*
  • Carcinoma in Situ / drug therapy*
  • Carcinoma in Situ / pathology
  • Conjunctival Neoplasms / drug therapy*
  • Conjunctival Neoplasms / pathology
  • Corneal Diseases / drug therapy*
  • Corneal Diseases / pathology
  • Cross-Over Studies
  • Double-Blind Method
  • Eye Neoplasms / drug therapy*
  • Eye Neoplasms / pathology*
  • Female
  • Follow-Up Studies
  • Humans
  • Male
  • Middle Aged
  • Mitomycin / administration & dosage*
  • Prospective Studies
  • Treatment Outcome


  • Antibiotics, Antineoplastic
  • Mitomycin