Topical 5-fluorouracil 1% as first-line therapy for giant ocular surface squamous neoplasia.

PURPOSE

To evaluate the clinical outcomes of topical 5-fluorouracil (5-FU) 1% as first-line therapy for giant ocular surface squamous neoplasia (OSSN).

MATERIALS AND METHODS

This was a non-comparative cohort study. We included patients with biopsy-proven giant OSSN in a tertiary-care setting. Giant OSSN was defined as a single lesion≥15mm in the largest basal dimension or an extent of≥6 clock hours of limbal involvement. Topical 5-FU 1% was administered QID for one week, followed by a three-week drug holiday. This treatment cycle was repeated until resolution. Resolution rate was the primary outcome measure, where complete resolution was defined clinically and by tomography. Secondary outcomes were time to resolution and frequency of recurrence and side effects.

RESULTS

Twenty-seven eyes (27 patients) were included; the majority (19, 70%) were men, with a mean age of 69.2±15.7 years. Corneal/conjunctival intraepithelial neoplasia was diagnosed in 88%, and squamous cell carcinoma (SCC) in the remaining 12% of cases. The rate of complete resolution was 77.8% (21/27) after a mean of 5.1±2.1 cycles. The median time to complete resolution was 5 months (CI95% 4-6 months). During a mean follow-up of 10.2±2.9 months, recurrence was observed in one patient (4.8%) and four (14.8%) reported side effects. Patients with partial resolution were older and had a higher frequency of SCC diagnosis than those with complete resolution.

CONCLUSION

Topical 5-FU 1% appears to be useful as first-line therapy for giant OSSN, with good tolerance and a low frequency of recurrence. More studies with larger sample sizes and longer follow-up are needed.