12-month outcomes after voretigene neparvovec gene therapy in paediatric patients with

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Tác giả: Emilie Batté, Dominique Bremond Gignac, Alejandra Daruich, Nathalie de Vergnes, Maxence Rateaux, Matthieu P Robert, Sophie Valleix

Ngôn ngữ: eng

Ký hiệu phân loại: 615.895 Gene therapy

Thông tin xuất bản: England : The British journal of ophthalmology , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 677826

AIMS: To report main outcomes and complications following voretigene neparvovec (Luxturna) treatment in paediatric patients. METHODS: Records of patients under the age of 17 treated by subretinal administration of voretigene neparvovec for confirmed biallelic RESULTS: 12 eyes of six patients (mean age: 7.8 years) were analysed. No intraoperative complications occurred. BCVA significantly improved at 12-month follow-up (mean LogMAR (logarithm of the minimal angle of resolution) BCVA: 1.0±0.8 at baseline vs 0.6±0.3 at 12 months, p=0.001). Mean central macular thickness and central outer nuclear layer thickness did not change at 12 months follow-up. VF V4e isopter did not show significant changes. Postoperatively complications included: elevated intraocular pressure in two eyes of the same patient, a parafoveal lamellar hole at 3 months post-treatment and atrophy on the injection site observed in all eyes except one, which significantly enlarged during 12 months (p=0.008). CONCLUSIONS: Most paediatric patients treated by voretigene neparvovec showed a significant increase in visual function at 12 months follow-up. None of the postoperative complications prevented gains in visual function.
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