Purpose
To determine the efficacy of topical 5-fluorouracil 1% (5FU) as a primary treatment of ocular surface squamous neoplasia (OSSN).
Design
Retrospective study.
Participants
Topical 5FU was used as primary therapy in 44 patients with OSSN.
Methods
5FU 1% administered topically four times a day for a week followed by a drug holiday of 3 weeks. Patients were identified through pharmacy database. Patients were excluded if 5FU was used as adjuvant therapy, did not complete therapy, or were still in active treatment for their OSSN at the time of last follow up.
Main outcome measure
The primary outcome measures were the frequency of complete resolution with topical 5FU treatment and the rate of OSSN recurrence.
Results
Of the 44 patients identified, 32 were males and 12 were females. The mean age was 68 years. Complete resolution of OSSN was noted in 82% (36/44) patients; and, 18% (8/44) were considered treatment non-responders. Patients were treated a median of 4 cycles (range 2-9). Nasal location was the only risk factor identified for nonresponse to therapy (p=0.04). The median follow up after resolution was 10 months (range 2-77). In the 36 patients with complete resolution, 4 had tumor recurrence. Recurrence rates at 1 and 2 years were 6% and 15% respectively using Kaplan-Meier survival analysis. At least one side effect from the medication was reported by 61% (21/44) of patients, but only 1 patient discontinued the medication due to intolerance. The most common side effect was pain (n=17, 39%) followed by tearing (n=10, 23%), photophobia (n=6, 14%), itching (n=4, 9%), swelling (n=2, 5%), and infection (n=1, 2%). No long-term complications were reported.
Conclusion
5-fluorouracil is efficacious and well tolerated as a primary treatment for OSSN, with 82% of tumors completely responding to therapy.