Evidence map›Paper›PMID 33972235›Full record

ArticleThe British journal of ophthalmology2022

Evaluation of intravitreal topotecan dose levels, toxicity and efficacy for retinoblastoma vitreous seeds: a preclinical and clinical study.

Carley M Bogan, Jessica V Kaczmarek, Janene M Pierce, Sheau-Chiann Chen, Kelli L Boyd, Marion W Calcutt, Thomas M Bridges, Craig W Lindsley, Jennifer B Nadelmann, Albert Liao and 6 more

Registry-linked trialOpen access · hybridAbstract read
In one paragraph

Article in The British journal of ophthalmology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06425419 (The Safety and Efficacy of Intravitreal Topotecan for the Treatment of Proliferative Vitreoretinopathy), which is not on this map. Cited by 20 papers.

0numbers the graph read from it
0cells of the map it votes in
20citing papers in PubMed
5.1field-weighted citation impact, top 4% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT06425419 phase1not yet recruitingnot on this mapstarted 2026, after this paper: background citation

The Safety and Efficacy of Intravitreal Topotecan for the Treatment of Proliferative Vitreoretinopathy

TypeinterventionalSponsorMassachusetts Eye and Ear InfirmaryRan2026 to 2028Enrolled50ConditionsProliferative Vitreoretinopathy, Proliferative Vitreo-Retinopathy, Rhegmatogenous Retinal DetachmentArmsTopotecan
3 · Its place in the literature

Who cites it

20 citing papers in PubMed, 39 citations in OpenAlex.

  1. Topotecan Synergizes with SP141 in the Management of Uveal Melanoma.bioRxiv : the preprint server for biology · 2026
    Article
  2. Article
  3. Article
  4. Stability study of topotecan in ophthalmic prefilled syringes for intravitreal delivery.European journal of hospital pharmacy : science and practice · 2025
    Article
  5. Article
  6. Article
  7. Article
  8. Update on Retinoblastoma Therapies.Medicina (Kaunas, Lithuania) · 2025
    Review
  9. Review
  10. Histone Deacetylases in Retinoblastoma.International journal of molecular sciences · 2024
    Review
  11. Article
  12. Review
  13. Emerging New Therapeutics for Retinoblastoma.Ocular oncology and pathology · 2022
    Review
  14. Article
  15. Article
  16. Review
  17. Article
  18. Article
  19. Article
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

16 authors at 3 institutions in 1 country.

Carley M BoganOphthalmology & Visual Sciences, Vanderbilt University Medical Center, Nashville, Tennessee, USA.
Jessica V KaczmarekOphthalmology & Visual Sciences, Vanderbilt University Medical Center, Nashville, Tennessee, USA.
Janene M PierceOphthalmology & Visual Sciences, Vanderbilt University Medical Center, Nashville, Tennessee, USA.
Sheau-Chiann ChenCenter for Quantitative Sciences, Vanderbilt University Medical Center, Nashville, Tennessee, USA.
Kelli L BoydVanderbilt-Ingram Cancer Center, Vanderbilt University Medical Center, Nashville, Tennessee, USA.
Marion W CalcuttBiochemistry, Vanderbilt University, Nashville, Tennessee, USA.
Thomas M BridgesCenter for Neuroscience Drug Discovery, Vanderbilt University, Nashville, Tennessee, USA.
Craig W LindsleyCenter for Neuroscience Drug Discovery, Vanderbilt University, Nashville, Tennessee, USA.
Jennifer B NadelmannSurgery, Memorial Sloan Kettering Cancer Center, New York, New York, USA.ORCID 0000-0002-7021-9986
Albert LiaoSurgery, Memorial Sloan Kettering Cancer Center, New York, New York, USA.
Terry HsiehSurgery, Memorial Sloan Kettering Cancer Center, New York, New York, USA.
David H AbramsonSurgery, Memorial Sloan Kettering Cancer Center, New York, New York, USA.
Jasmine H FrancisSurgery, Memorial Sloan Kettering Cancer Center, New York, New York, USA.ORCID 0000-0002-1889-3447
Debra L FriedmanVanderbilt-Ingram Cancer Center, Vanderbilt University Medical Center, Nashville, Tennessee, USA.
Ann RichmondVanderbilt-Ingram Cancer Center, Vanderbilt University Medical Center, Nashville, Tennessee, USA.
Anthony B DanielsOphthalmology & Visual Sciences, Vanderbilt University Medical Center, Nashville, Tennessee, USA anthony.b.daniels@vumc.org.ORCID 0000-0001-8361-9885
Vanderbilt University Medical Center · USMemorial Sloan Kettering Cancer Center · USVanderbilt University · US

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Hongbiao Carl Lekaye · 1985 to 2026
$347.4M
Tumor Immunology and Microenvironment Research ProgramP30CA068485 · NCI · VANDERBILT UNIVERSITY MEDICAL CENTER · PI STACIE B DUSETZINA · 1995 to 2026
$172.8M
VANDERBILT UNIVERSITY CTSA FOR PEDIATRIC RESEARCHUL1RR024975 · NCRR · VANDERBILT UNIVERSITY · PI BERNARD, GORDON RAPHAEL · 2007 to 2011
$45.7M
The Vanderbilt Institute for Clinical and Translational Research (VICTR)UL1TR000445 · NCATS · VANDERBILT UNIVERSITY MEDICAL CENTER · PI BERNARD, GORDON RAPHAEL · 2012 to 2016
$41.4M
Training (pp 214-245)P41GM103391 · NIGMS · VANDERBILT UNIVERSITY · PI CAPRIOLI, RICHARD M · 2012 to 2020
$17.7M
Developing alternative approaches to reduce retinal toxicity and prevent vision loss in the treatment of intraocular retinoblastomaK08EY027464 · NEI · VANDERBILT UNIVERSITY MEDICAL CENTER · PI DANIELS, ANTHONY BRENT · 2017 to 2021
$960k
BLR&D Merit Review Research Career Scientist (RCS) Award (IK6)IK6BX005225 · VA · VETERANS HEALTH ADMINISTRATION · PI RICHMOND, ANN · 2020 to 2023
–
BLRD VA IK6 BX005225NCATS NIH HHS UL1 TR000445NCI NIH HHS P30 CA008748NCI NIH HHS P30 CA068485NCRR NIH HHS UL1 RR024975NEI NIH HHS K08 EY027464NIGMS NIH HHS P41 GM103391
6 · The paper itself

Abstract

backgroundCurrent melphalan-based intravitreal regimens for retinoblastoma (RB) vitreous seeds cause retinal toxicity. We assessed the efficacy and toxicity of topotecan monotherapy compared with melphalan in our rabbit model and patient cohort.

methodsRabbit experiments: empiric pharmacokinetics were determined following topotecan injection. For topotecan (15 μg or 30 µg), melphalan (12.5 µg) or saline, toxicity was evaluated by serial electroretinography (ERG) and histopathology, and efficacy against vitreous seed xenografts was measured by tumour cell reduction and apoptosis induction. PATIENTS: retrospective cohort study of 235 patients receiving 990 intravitreal injections of topotecan or melphalan.

resultsIntravitreal topotecan 30 µg (equals 60 µg in humans) achieved the IC

conclusionsTaken together, these experiments suggest that intravitreal topotecan monotherapy for the treatment of RB vitreous seeds is non-toxic and effective.

Indexed as

Retinal NeoplasmsRetinoblastomaAnimalsAntineoplastic Agents, AlkylatingHumansIntravitreal InjectionsMelphalanNeoplasm SeedingRabbitsRetrospective StudiesTopotecanVitreous BodyAntineoplastic Agents, AlkylatingMelphalanTopotecananimal modelsefficacyintravitreal chemotherapypharmacokineticsretinoblastomatopotecantoxicityvitreous seeds

Identifiers

PMID33972235
PMCPMC8788260
OpenAlexW3161935658

What OpenQuestion holds

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LicenceCC BY-NC
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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.