Topical 1% 5-fluorouracil eye drops as primary treatment for ocular surface squamous neoplasia: Long-term follow-up study

Adam Wylegala; Wathanee Sripawadkul; Mike Zein; Osmel P Alvarez; Ghada Al Bayyat; Anat Galor; Carol L Karp

doi:10.1016/j.jtos.2022.12.002

Topical 1% 5-fluorouracil eye drops as primary treatment for ocular surface squamous neoplasia: Long-term follow-up study

Ocul Surf. 2023 Jan:27:67-74. doi: 10.1016/j.jtos.2022.12.002. Epub 2022 Dec 5.

Authors

Adam Wylegala¹, Wathanee Sripawadkul², Mike Zein³, Osmel P Alvarez³, Ghada Al Bayyat³, Anat Galor⁴, Carol L Karp⁵

Affiliations

¹ Bascom Palmer Eye Institute, Department of Ophthalmology, University of Miami, Miami, FL, USA; Health Promotion and Obesity Management Unit, Department of Pathophysiology, Faculty of Medical Sciences in Katowice, Medical University of Silesia, Poland.
² Bascom Palmer Eye Institute, Department of Ophthalmology, University of Miami, Miami, FL, USA; Department of Ophthalmology, Faculty of Medicine, Srinakharinwirot University, Bangkok, Thailand.
³ Bascom Palmer Eye Institute, Department of Ophthalmology, University of Miami, Miami, FL, USA.
⁴ Bascom Palmer Eye Institute, Department of Ophthalmology, University of Miami, Miami, FL, USA; Department of Ophthalmology, Miami Veterans Administration Medical Center, Miami, FL, USA.
⁵ Bascom Palmer Eye Institute, Department of Ophthalmology, University of Miami, Miami, FL, USA. Electronic address: ckarp@med.miami.edu.

PMID: 36476665
DOI: 10.1016/j.jtos.2022.12.002

Abstract

Purpose: To determine the efficacy and safety of topical 1% 5-fluorouracil (5FU) eye drops as primary treatment of ocular surface squamous neoplasia (OSSN).

Methods: Patients were diagnosed with OSSN based on slit-lamp examination and anterior segment optical coherence tomography (AS-OCT). In ambiguous cases an incisional biopsy was performed. All were treated with 5FU eye drops as primary therapy and retrospectively reviewed. Data on demographics, tumor characteristics, treatment outcome, and side effects were collected. The primary outcome measures were resolution frequency and recurrence rate of OSSN. Secondary outcomes were risk factors for resolution and recurrence, and side effects of treatment.

Results: The mean age of 251 subjects (258 eyes) was 67.5 ± 11.7 years, 182 were male. Patients were followed up on average for 752 ± 580 days. Overall, 87% of patients experienced complete tumor resolution. Multivariable analysis revealed that superior tumor location (HR: 0.62, 95% CI: 0.41-0.93, p = 0.02) and leukoplakia (HR: 0.65, 95% CI: 0.41-0.93, p = 0.02), decreased the likelihood of tumor resolution. The recurrence rate was 4% at six months, 8% at one year, and 19% at two years. Larger tumor area increased chances of tumor recurrence (HR: 1.01, 95% CI: 1.00-1.02 p = 0.03). The most common side effects of 5-FU were mild hyperemia and pain, which occurred in 26% and 23% of patients, respectively. Among the sight-threatening side effects, limbal stem cell deficiency occurred in only 3% of patients.

Conclusions: Topical 1% 5FU eye drops are a safe and effective medication for OSSN. Superior tumor location and leukoplakia decreased the chance of tumor resolution.

Keywords: IFN; MMC; OCT; Ocular oncology; Squamous cell carcinoma.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

Carcinoma, Squamous Cell* / diagnosis
Carcinoma, Squamous Cell* / drug therapy
Carcinoma, Squamous Cell* / pathology
Conjunctival Neoplasms* / drug therapy
Conjunctival Neoplasms* / pathology
Eye Neoplasms* / diagnosis
Eye Neoplasms* / drug therapy
Eye Neoplasms* / pathology
Female
Fluorouracil / therapeutic use
Follow-Up Studies
Humans
Male
Neoplasm Recurrence, Local / drug therapy
Ophthalmic Solutions / therapeutic use
Retrospective Studies

Substances

Ophthalmic Solutions
Fluorouracil

Abstract

Publication types

MeSH terms

Substances

Grants and funding