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The Capital Region of Denmark - a part of Copenhagen University Hospital
E-pub ahead of print

Inflammation after voretigene neparvovec administration in patients with RPE65-related retinal dystrophy

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OBJECTIVE: To report on the prevalence of intraocular inflammation after subretinal voretigene neparvovec (VN) administration.

DESIGN: Retrospective review of medical files.

PARTICIPANTS: All patients receiving VN in Denmark.

METHODS: Twelve patients had received VN gene therapy as standard-of-care for bi-allelic RPE65-related retinal disease. Bilateral treatment had been performed in 11 patients and unilateral treatmen in one patient. Patients had been followed clinically before and after VN administration using functional measurements (visual acuity, full-field scotopic threshold (FST) test, visual fields) and structural evaluations (fundus imaging (color and autofluorescence), optical coherence tomography (OCT), slitlamp).

MAIN OUTCOMES: Signs of intraocular inflammation including vitritis and outer retinal infiltrates.

RESULTS: Vitritis was observed in 9 out of 23 eyes receiving VN. The median time to resolution of vitritis from the time of treatment was 89 days. Four eyes also presented with outer retinal infiltrates at the time of vitritis. Inflammation subsided on immunosuppressant therapy. The presence of inflammation did not adversely affect visual outcome after VN therapy. In one eye outer retinal infiltrates were demonstrated to precede later development of atrophy.

CONCLUSION: Patients undergoing subretinal gene therapy needs to be closely monitored for signs of inflammation and although we did not observe a detrimental effect on visual function in eyes with inflammation, it seems wise to treat it appropriately as it may lead to atrophy of the RPE and outer retina. Also, it seems advisable to reduce the inflammatory load such as using a surgical technique that minimizes residual viral vectors in the vitreous body.

Original languageEnglish
JournalOphthalmology
ISSN0161-6420
DOIs
Publication statusE-pub ahead of print - 24 Jun 2022

Bibliographical note

Copyright © 2022. Published by Elsevier Inc.

ID: 79043858