High-Dose Intravitreal Topotecan Combined With Focal Consolidation Therapy for Recurrent Retinal Tumors in Retinoblastoma

Abstract

Purpose: To evaluate tumor control and ocular safety associated with high-dose intravitreal (IVT) topotecan (100 µg) for isolated recurrent solid retinal tumors in children with retinoblastoma. Methods: This retrospective case series was based on a prospectively maintained clinical database and included 10 eyes of 10 patients with isolated recurrent solid retinal tumors following prior systemic and/or intra-arterial chemotherapy. All eyes received IVT topotecan (100 µg/0.1 mL) using a standardized injection protocol, with adjunctive focal consolidation therapy (laser photocoagulation and/or cryotherapy) administered as clinically indicated. Primary outcomes included complete tumor regression, globe salvage, ocular toxicity, and metastatic events. Results: The mean interval from the last prior treatment to recurrence was 13 (range, 3–24) months. Each eye received a median of 2 injections (range, 1–6; mean, 2.7 injections). Complete tumor regression was achieved in all eyes (100%). No clinically appreciable ocular toxicity, systemic toxicity, enucleations, or metastatic events occurred during a mean follow-up of 12 (range, 6–24) months. Conclusions: High-dose IVT topotecan combined with focal consolidation therapy appears to be a promising treatment option for isolated recurrent solid retinal tumors in retinoblastoma, achieving excellent tumor control with low observed toxicity during the available follow-up period.