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Related Experiment Video

Updated: Dec 14, 2025

Subretinal Transplantation of Human Embryonic Stem Cell Derived-retinal Pigment Epithelial Cells into a Large-eyed Model of Geographic Atrophy
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Is intravitreal topotecan toxic to retinal function?

Jennifer Nadelmann1,2, Jasmine H Francis3,4, Scott E Brodie5

  • 1Ophthalmic Oncology Service, Memorial Sloan Kettering Cancer Center, New York, New York, USA jennifer.nadelmann@gmail.com.

The British Journal of Ophthalmology
|July 16, 2020
PubMed
Summary

Intravitreal topotecan combined with melphalan may cause retinal toxicity in retinoblastoma patients. However, topotecan injections alone at standard doses did not show significant retinal toxicity based on electroretinogram results.

Keywords:
ElectrophysiologyNeoplasiaPharmacologyRetinaTreatment Medical

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Area of Science:

  • Ophthalmology
  • Oncology
  • Retinal Research

Background:

  • Intravitreal topotecan is a treatment for retinoblastoma with vitreous seeds.
  • The potential for retinal toxicity with intravitreal topotecan requires evaluation.

Purpose of the Study:

  • To assess the association between intravitreal topotecan and retinal toxicity in retinoblastoma patients.
  • To determine if topotecan, alone or in combination, impacts retinal function.

Main Methods:

  • Retrospective cohort study of 28 eyes treated with intravitreal topotecan between 2014-2019.
  • Electroretinograms (ERGs) measured pre-treatment and one month post-injection.
  • Ocular toxicity defined by a decrease in 30 Hz ERG amplitude.

Main Results:

  • Concurrent intravitreal topotecan and melphalan (22 injections) showed a significant decrease in ERG amplitude (p=0.046).
  • Intravitreal topotecan alone (28 injections) at doses of 20-30 μg did not result in a significant ERG amplitude decrease (p=0.85).
  • Median follow-up for ERG measurement was 27 days.

Conclusions:

  • Intravitreal topotecan in combination with melphalan may be associated with retinal toxicity.
  • Intravitreal topotecan monotherapy at doses of 20-30 μg appears safe regarding retinal toxicity in retinoblastoma.
  • ERG is a valuable tool for assessing treatment-related ocular toxicity.