Evidence map›Paper›PMID 41379738›Full record

ArticleOphthalmologica. Journal international d'ophtalmologie. International journal of ophthalmology. Zeitschrift fur Augenheilkunde2026

Chorioretinal Atrophy after Voretigene Neparvovec Therapy in <italic>RPE65</italic> Mutation-Associated Inherited Retinal Disease: Longitudinal Characterization in Multimodal Imaging.

Sandrine H Sassen, Marlene Sassmannshausen, Josua Sassen, Markus N Preising, Johanna P Scholz, Frank G Holz, Birgit Lorenz, Philipp Herrmann

Abstract read
In one paragraph

Article in Ophthalmologica. Journal international d'ophtalmologie. International journal of ophthalmology. Zeitschrift fur Augenheilkunde, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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.

Sandrine H SassenDepartment of Ophthalmology, University of Bonn, Bonn, Germany.
Marlene SassmannshausenDepartment of Ophthalmology, University of Bonn, Bonn, Germany.
Josua SassenInstitute or Numerical Simulation, University of Bonn, Bonn, Germany.
Markus N PreisingDepartment of Ophthalmology, Justus-Liebig-University Giessen, Giessen, Germany.
Johanna P ScholzDepartment of Ophthalmology, University of Bonn, Bonn, Germany.
Frank G HolzDepartment of Ophthalmology, University of Bonn, Bonn, Germany.
Birgit LorenzDepartment of Ophthalmology, University of Bonn, Bonn, Germany.
Philipp HerrmannDepartment of Ophthalmology, University of Bonn, Bonn, Germany, philipp.herrmann@ukbonn.de.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

<p>Introduction: The detection of chorioretinal atrophy (CRA) following voretigene neparvovec (VN) therapy for RPE65-IRD highlights the need for long-term monitoring to better understand the safety and efficacy of this gene augmentation treatment. This study aimed to longitudinally assess the development of VN-associated CRA, its presentation in multimodal retinal imaging, and potential implications on visual function in patients with RPE65-IRD.

methodsThis single-center, prospective cohort study was conducted at the University of Bonn. A total of 21 patients with confirmed RPE65-IRD underwent subretinal VN therapy. Multimodal imaging, including blue-light fundus autofluorescence (BAF), near-infrared imaging (IR), near-infrared fundus autofluorescence (IRAF), and color fundus photography (CFP), assessed atrophy development. The primary outcome measure was the incidence and size of CRA, while secondary outcomes included the relationship between CRA and changes in visual function.

resultsThe study reported a 50% (16/32 eyes) incidence of CRA, with lesions primarily located in the macula (86.7%), bleb area (86.67%), injection site (80%), and periphery (80%). Lesion detection and size varied between BAF, IR, IRAF, and CFP, with the largest CRA detected in BAF imaging. Initially, the median enlargement rate of CRA was 60.50 mm2/year (131.20). Notably, eyes with CRA development had significantly better baseline low luminance visual acuity. DISCUSSION: This study highlights the crucial role of multimodal imaging in monitoring VN-associated CRA. Differences in lesion detection and size assessment across imaging modalities were observed, with the largest CRA extent detected in BAF imaging. Median visual function remained stable. These findings emphasize the complexity of VN therapy outcomes and the need for close surveillance using combined multimodal imaging to better understand the long-term clinical implications. </p>.

Indexed as

Choroidcis-trans-IsomerasesDNAFluorescein AngiographyGenetic TherapyMultimodal ImagingMutationTomography, Optical CoherenceVisual AcuityAdolescentAdultChildFemaleFollow-Up StudiesFundus OculiHumanscis-trans-IsomerasesDNAChorioretinal atrophyInherited retinal diseaseMultimodal retinal imagingRPE65-IRDSubretinal gene augmentation therapyVoretigene neparvovec

Identifiers

PMID41379738
PMCPMC12788828

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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.