Article Text

Download PDFPDF
Is intravitreal topotecan toxic to retinal function?
  1. Jennifer Nadelmann1,2,
  2. Jasmine H Francis1,3,
  3. Scott E Brodie4,
  4. Engjel Muca1,
  5. David H Abramson1,3
  1. 1 Ophthalmic Oncology Service, Memorial Sloan Kettering Cancer Center, New York, New York, USA
  2. 2 Scheie Eye Institute, Philadelphia, Pennsylvania, USA
  3. 3 Department of Ophthalmology, Weill Cornell Medical College, New York, New York, USA
  4. 4 NYU Langone Health, New York, New York, USA
  1. Correspondence to Jennifer Nadelmann, Scheie Eye Institute, 51 North 39th Street, Philadelphia, PA 19104, USA; jennifer.nadelmann{at}


Background Intravitreal injections of topotecan are used in the management of retinoblastoma with vitreous seeds. This study evaluated whether intravitreal topotecan was associated with retinal toxicity.

Methods Retrospective cohort study of patients with retinoblastoma who were treated with intravitreal topotecan at Memorial Sloan Kettering Cancer Center between December 2014 and May 2019. Electroretinogram (ERG) responses under anaesthesia were measured immediately before treatment with intravitreal topotecan and at the next visitor approximately one-month. Ocular toxicity was defined by a decrease in the ERG response at 30 Hz at follow-up.

Results Ocular toxicity was evaluated by ERG on 50 evaluable injections administered to 28 eyes. 22 (44.0%) injections were performed with concurrent intravitreal melphalan. The median time to ERG measurement following an injection was 27 days. By using a paired t-test, intravitreal topotecan combined with melphalan (n=22) at a dose of 25 μg or 30 μg was associated with a significant decrease in ERG amplitude at follow-up (p=0.046, 95% CI −20.4 μV to −0.2 μV). Among eyes that only received topotecan (n=28) at doses of 20 μg or 30 μg, there was not a significant difference in ERG amplitude measured (p=0.85, 95% CI −7.0 μV to 5.8 μV).

Conclusion Intravitreal topotecan combined with intravitreal melphalan was associated with a decrease in ERG amplitude; there was not a significant decrease in ERG amplitude observed in patients who received topotecan alone. These findings suggest that intravitreal topotecan injections at doses of 20 μg or 30 μg are not associated with retinal toxicity in patients with retinoblastoma.

  • Treatment Medical
  • Retina
  • Pharmacology
  • Neoplasia
  • Electrophysiology

Statistics from

Request Permissions

If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.


  • Contributors Conception or design of the work: JN, DHA, JHF. Data collection: JN, DHA, JHF, SEB, EM. Data analysis and interpretation: JN, SEB, DHA, JHF. Drafting the article: JN, DHA, JHF. Critical revision of the article: JN, DHA, JHF, SEB, EM.

  • Funding Supported in part by grants from the Fund for Ophthalmic Knowledge, New York, New York and Cancer Center Support Grant (P30 CA008748). The sponsor or funding organization had no role in the design or conduct of this research.

  • Competing interests None declared.

  • Provenance and peer review Not commissioned; externally peer reviewed.

  • Data availability statement Data are available upon reasonable request.

Linked Articles

  • At a glance
    Frank Larkin