High-Dose Intravitreal Topotecan for Retinoblastoma With Subretinal Seeds: A Well-Tolerated and Efficacious Approach.

Source: PubMed, NCBI / U.S. National Library of Medicine

High-dose intravitreal topotecan (90 μg) has emerged as a promising treatment option for refractory or recurrent retinoblastoma with active tumor and subretinal seeds, offering localized disease control while minimizing systemic toxicity. In this 5-patient case series, tumor regression was achieved in 4 cases without significant adverse effects. One patient required enucleation due to disease progression. Timely administration and consistent follow-up were identified as key factors influencing treatment outcomes. High-dose intravitreal topotecan demonstrates meaningful therapeutic potential in achieving localized tumor control with an acceptable safety profile, supporting further investigation into its role in managing refractory retinoblastoma.