Ocular Surface Squamous Neoplasia: A Cohort Study and Updates in the Pathogenesis, Diagnosis, and Medical Management

To review the current literature on the pathogenesis, diagnosis, and management of ocular surface squamous neoplasia (OSSN), and to evaluate the efficacy and safety of topical therapies—interferon alpha-2b (IFNα-2b), mitomycin-C (MMC), and 5-fluorouracil (5-FU)—in a retrospective cohort of patients with OSSN. A comprehensive literature review was conducted alongside a retrospective cohort study involving 43 patients diagnosed with OSSN. Treatment outcomes, recurrence rates, and toxicity profiles associated with IFNα-2b, MMC, and 5-FU were analyzed. Data on treatment duration, number of cycles required for tumor resolution, and associations with patient demographics were assessed. A mean of 2.56 treatment cycles was required for complete tumor resolution across the cohort. IFNα-2b led to tumor resolution in a shorter time frame compared to 5-FU. No significant correlation was identified between patient demographics and treatment response. However, a history of cancer was associated with larger tumor size and greater limbal involvement at presentation. Topical agents are effective and well-tolerated treatments for OSSN, with IFNα-2b demonstrating faster response times in this cohort. The literature highlights a lack of prospective randomized trials comparing these therapies. Furthermore, limited understanding of the molecular mechanisms underlying OSSN pathogenesis continues to hinder individualized treatment strategies and risk stratification.