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

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.