Topical Chemotherapy for Ocular Surface Squamous Neoplasia: A Review of Adverse Effects and Their Clinical Management.

Abstract

Topical chemotherapy is increasingly used to treat ocular surface tumors as a primary therapy and an adjuvant treatment after surgical excision. The most employed topical agents include mitomycin C (MMC), 5-fluorouracil (5-FU), and interferon alpha-2b (IFNα2b), each with distinct mechanisms of action, efficacy profiles, and toxicity risks. Although these agents offer effective tumor control and allow for a non-invasive approach in many cases, ocular surface complications requiring medical or surgical management can occur. This summarizes the adverse effect and outilines practical strategies for their prevention and treatment. MMC is the most potent agent but also the most toxic, with reported complications such as limbal stem cell deficiency, punctal stenosis, and persistent epithelial defects. 5-FU demonstrates a more favorable safety profile, although rare cases of corneal ulceration have been described. IFNα2b is well tolerated and associated primarily with mild, reversible reactions. The choice of the proper agent should be tailored according to patient's clinical presentation, ocular surface status, and ability to adhere to therapy and follow-up. Timely recognition and management of complications are essential to minimize long-term sequelae. Reliance on compounded formulations highlights the need for stable, standardized, and commercially available topical agents specifically designed for ocular use to ensure safety, reproducibility, and global accessibility.