Topical 5-Fluorouracil 1% As First-Line Therapy For Giant Ocular Surface Squamous Neoplasia: Short-Term Outcomes

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

Single center, Cornea and refractive surgery Department at Asociación para evitar la ceguera en México I.A.P

In this retrospective cohort study, we reviewed the medical records of patients treated for biopsy-proven giant OSSN. All patients received 5-FU four times daily for one week, followed by a three-week drug holiday. Giant OSSN was defined as a single lesion with ≥ 15 mm in the largest basal diameter or extent ≥ 6 limbal clock-hours. The resolution rate was the primary outcome measure. Complete resolution was defined as the clinical and tomographic absence of lesions. Secondary outcomes were time to resolution, frequency of recurrences, and side effects.

Twenty-five eyes (25 patients) diagnosed with giant OSSN were included. Most of them (17, 68%) were men, with a mean age of 70.4 ± 14.6 years. Complete resolution was achieved in 19 cases (76%). The median time to complete resolution was 5 months ( CI 95% 4-6 months), after a mean of 4.9 ± 2.1 cycles. During a mean follow-up of 10.2 ± 3.1 months, recurrence was observed in 1(5.3%) patient, and 4(16%) patients reported side effects. Cases with partial resolution were older and had a higher frequency of squamous cell carcinoma (SCC) diagnosis than those with complete resolution.

Topical 5-fluorouracil 1% appears to be useful as first-line therapy for giant OSSN, with good tolerance and low frequency of recurrence. This is the largest series reported that assess the effect of 5-FU for extensive tumors; however, prospective studies with larger sample size and longer follow-up are needed to determine the treatment efficacy.