High-Dose Topotecan for Recurrent Retinal and Subretinal Retinoblastoma

Background/Objectives: To report on the efficacy and toxicity of high-dose intravitreal topotecan (90–180 µG) as treatment for recurrent retinal and subretinal retinoblastoma. Methods: We conducted a retrospective review of children with recurrent retinoblastoma managed with intravitreal topotecan 90–180 µG in a single tertiary cancer center (Memorial Sloan Kettering Cancer Center). Toxicity was based on physical exam and serial 30-Hz electroretinograms (ERGs). Results: In our analysis, we included 49 patients (average age, 42 months) who received 114 intravitreal injections of topotecan 90–180 µG for recurrent retinal and/or subretinal tumors. Of the 114 injections in 81 treated eyes, 75 included concurrent indirect 810-nm diode laser. Overall, 83 injections caused a complete response and 31 caused a partial response. All patients are alive (mean follow-up, 14 months), and one eye came to enucleation. There were no adverse retinal events, including no extraocular extension, and ERGs (30 Hz) were unchanged at last follow-up. Conclusions: High-dose intravitreal topotecan (90–180 µG) was effective in controlling recurrent retinal and subretinal retinoblastoma in 48 of 49 patients without retinal or ERG toxicity.