Inflammation after Voretigene Neparvovec Administration in Patients with RPE65-Related Retinal Dystrophy

Research output: Contribution to journalJournal articlepeer-review

Purpose: 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 received VN gene therapy as standard of care for biallelic RPE65-related retinal disease. Bilateral treatment was performed in 11 patients and unilateral treatment in 1 patient. Patients were followed clinically before and after VN administration using functional measurements (visual acuity, full-field scotopic threshold test, visual fields) and structural evaluations (fundus imaging [color and autofluorescence], OCT, slit-lamp). Main Outcome Measures: Signs of intraocular inflammation, including vitritis and outer retinal infiltrates. Results: Vitritis was observed in 9 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 1 eye, outer retinal infiltrates were demonstrated to precede later development of atrophy. Conclusions: Patients undergoing subretinal gene therapy need to be closely monitored for signs of inflammation. Although we did not observe a detrimental effect on visual function in eyes with inflammation, it seems wise to treat it appropriately because it may lead to atrophy of the retinal pigment epithelium 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
Volume129
Issue number11
Pages (from-to)1287-1293
Number of pages7
ISSN0161-6420
DOIs
Publication statusPublished - 2022

Bibliographical note

Publisher Copyright:
© 2022 American Academy of Ophthalmology

    Research areas

  • Gene therapy, Inherited retinal dystrophy, RPE65, Voretigene neparvovec

ID: 323573048