ArticleRetina (Philadelphia, Pa.)2025
HIGH-DOSE INTRAVITREAL TOPOTECAN (100 µ G/0.1 CC) AS MONOTHERAPY FOR RECURRENT/REFRACTORY INTRAOCULAR RETINOBLASTOMA IN SEVEN EYES.
Article in Retina (Philadelphia, Pa.), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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Who cites it
2 citing papers in PubMed.
- Retinoblastoma management: achieving high standards of care.Eye (London, England) · 2026Review
- Topotecan Microneedle Scleral Patch: A Transscleral Drug Delivery Study for Retinoblastoma.Ophthalmology science · 2026Article
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3 authors.
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Abstract
purposeTo report the outcomes and safety of 100 μ g/0.1 cc intravitreal topotecan (IVitT100) as monotherapy for refractory/recurrent intraocular retinoblastoma.
methodsRetrospective single-center study of 7 patients with retinoblastoma who received IVitT100 as monotherapy between January 2024 and December 2024 with a minimum follow-up of 3 months after the last injection.
resultsAt presentation, six eyes were classified as Group D and one as Group C, according to the International Classification of Retinoblastoma. High-dose topotecan was administered for refractory and recurrent disease in 2 (29%) and 5 (71%) eyes, respectively. The active disease was in the form of a intraretinal tumor with vitreous seeds (n = 3, 43%), intraretinal tumor with epiretinal seeds (n = 1, 14%), isolated intraretinal tumor (n = 1, 14%), isolated subretinal seeds (n = 1, 14%), or isolated vitreous seeds (n = 1, 14%). At a mean follow-up of 7 months (median, 6 months; range, 3-10) from the last injection of IVitT00, tumor control was achieved in 100% (4 of 4) eyes with vitreous seeds, 100% (1 of 1) with epiretinal seeds, 100% (1 of 1) with subretinal seeds, and 60% (3 of 5) with retinal tumor. Overall, tumor control was achieved in 5 eyes (71%) with IVitT100 monotherapy. Additional treatments were warranted in two eyes for complete tumor control. Globe salvage was achieved in all eyes (100%). The electroretinogram (n = 4) showed no significant changes compared with the baseline.
conclusionIVitT100 is a safe option for refractory or recurrent intraocular retinoblastoma and holds promise for controlling refractory vitreous seeds, subretinal seeds, and intraretinal tumors.
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