LACK OF COMPLICATIONS OF HIGH-DOSE INTRAVITREAL TOPOTECAN FOR RECURRENT RETINOBLASTOMA IN 81 CONSECUTIVE INJECTIONS.
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PURPOSE: To explore the safety of high-dose intravitreal topotecan for recurrent retinoblastoma management in human eyes. METHODS: There were 81 consecutive injections of high-dose intravitreal topotecan (90 μ g/0.18 cc‒100 µ g/0.20 cc on a monthly basis) in 25 eyes with recurrent retinoblastoma. Tumor control and injection-related complications were assessed at each visit. Each tissue was reviewed for complication-associated alterations, and systemic evaluation for myelosuppression, infection, metastasis, and death was assessed. RESULTS: At the time of injection, the mean patient age was 26 months, and in all cases, the injection was for recurrent retinoblastoma (n = 24 eyes, 100%) involving intraretinal tumor (n = 6 eyes, 24%), vitreous seeds (n = 3 eyes, 12%), subretinal seeds (n = 13 eyes, 52%), or multiple tumor types (n = 3 eyes, 12%). The mean intraretinal tumor thickness was 2.2 mm, vitreous seed thickness varied from pinpoint to confluent seeds, and subretinal seed was 0.9 mm. The total number of high-dose intravitreal topotecan injections was 81 (mean 3.2 per eye) with 39 (48%) injections given without concurrent chemotherapy and 42 (52%) given with concurrent intravenous or intra-arterial chemotherapy. At mean follow-up of 10 months after first injection, tumor control was achieved in all cases (n = 81 injections, 100%), and there was no local or systemic complication in any of the 81 injections. There was no case of extraocular tumor extension, myelosuppression, infection, metastasis, or death. CONCLUSION: Based on this analysis, high-dose intravitreal topotecan is safe and effective in the management of recurrent small retinoblastoma in humans. Further investigation of the limits of this therapy is warranted.