Evidence map›Paper›PMID 38322949›Full record

ArticleHeliyon2024

Could internal limiting membrane peeling before Voretigen neparvovec-ryzl subretinal injection prevent focal chorioretinal atrophy?

Lea Dormegny, Fouzia Studer, Arnaud Sauer, Laurent Ballonzoli, Claude Speeg-Schatz, Tristan Bourcier, Helene Dollfus, David Gaucher

Abstract read
In one paragraph

Article in Heliyon, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–field-weighted citation impact
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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed.

  1. Article
  2. Surgical Approaches to Retinal Gene Therapy: 2025 Update.Bioengineering (Basel, Switzerland) · 2025
    Review
  3. Long-term experience with gene augmentation therapy in patients with inherited retinal disease associated with biallelic mutations inMedizinische Genetik : Mitteilungsblatt des Berufsverbandes Medizinische Genetik e.V · 2025
    Article
  4. 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

8 authors.

Lea DormegnyDepartment of Ophthalmology, New Civil Hospital, Strasbourg University Hospital, Strasbourg, France.
Fouzia StuderInstitut de Génétique Médicale d'Alsace, CARGO Reference Center for Rare Diseases in Genetic Ophthalmology, University Hospital of Strasbourg, Strasbourg, France.
Arnaud SauerDepartment of Ophthalmology, New Civil Hospital, Strasbourg University Hospital, Strasbourg, France.
Laurent BallonzoliDepartment of Ophthalmology, New Civil Hospital, Strasbourg University Hospital, Strasbourg, France.
Claude Speeg-SchatzDepartment of Ophthalmology, New Civil Hospital, Strasbourg University Hospital, Strasbourg, France.
Tristan BourcierDepartment of Ophthalmology, New Civil Hospital, Strasbourg University Hospital, Strasbourg, France.
Helene DollfusInstitut de Génétique Médicale d'Alsace, CARGO Reference Center for Rare Diseases in Genetic Ophthalmology, University Hospital of Strasbourg, Strasbourg, France.
David GaucherDepartment of Ophthalmology, New Civil Hospital, Strasbourg University Hospital, Strasbourg, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Purpose: To report the effect of internal limiting membrane (ILM) peeling prior to Voretigen Neparvovec-ryzl (VN) subretinal injection on focal chorioretinal atrophy development in patients presenting with RPE65-mediated Leber congenital amaurosis (LCA). Design: Retrospective case series. Methods: Three patients who underwent bilateral subretinal VN injection for RPE65-mediated LCA were followed up for 18-24 months. ILM peeling was performed unilaterally in patients 1 and 2 and bilaterally in patient 3. Chorioretinal atrophy was identified on fundus biomicroscopy, non-mydriatic retinography and/or ultrawide field fundus imaging. Best corrected visual acuity (BCVA), spectral-domain optical coherence tomography (SD-OCT), visual fields, full-field stimulus threshold (FST) and visual functioning questionnaire score (NEI-VFQ-25) were reported. Outcome measures were changes in BCVA, visual fields, FST, NEI-VFQ-25, and chorioretinal atrophy location. Results: Chorioretinal atrophy at the injection site exclusively developed in eyes which did not undergo prior ILM peeling. In patient 3, bilateral pre-operative nummular chorioretinal alterations progressed toward epithelial atrophic patches in the mid and extreme retinal periphery 18 months after VN injection. BCVA and visual fields improved bilaterally. NEI_VFQ 25 remained stable in patient 1 and improved in patient 2 and 3. FST test improved bilaterally in patient 3. Conclusions: ILM peeling prior to VN injection seems to be a smoother and safer technique to administer VN treatment and may prevent secondary focal atrophy development at the injection site. However, another type of more extended chorioretinal atrophy might exist and could be related to LCA evolution or to incompletely understood adverse effect of VN product.

Indexed as

Gene therapyLeber congenital amaurosisLuxturnaPerifoveal chorioretinal atrophyRetinitis pigmentosaRPE65-mediated retinal dystrophy

Identifiers

PMID38322949
PMCPMC10844059

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Registered trials

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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.