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Real-world experience with Voretigene Neparvovec gene augmentation therapy in RPE65-mutation associated inherited retinal degeneration.

Ophthalmology 2023 September 12
OBJECTIVE: To assess the impact of baseline data on psychophysical and morphological outcomes of subretinal Voretigene Neparvovec (VN, Luxturna®) treatment.

DESIGN: Single center, retrospective, longitudinal, consecutive case series.

PARTICIPANTS: Patients with RPE65-IRD treated by one surgeon (FGH) between 02-2020 and 03-2022 with VN and oral immunosuppression according to the manufacturer´s recommendation.

METHODS: Retrospective analysis of surgical and clinical records, ancillary testing and retinal imaging following VN therapy for RPE65-IRD. Descriptive statistics compared data at baseline up to 24 months post treatment.

MAIN OUTCOME MEASURES: Best-corrected visual acuity (BCVA), low-luminance VA (LLVA), Goldmann Visual Fields (GVF), chromatic full-field-stimulus-threshold testing (FST), scotopic and photopic 2-Color-Threshold Perimetry (2CTP), multimodal retinal imaging.

RESULTS: 30 eyes / 19 patients were analyzed (10 pediatric <20y, 20 adult (range 8-40 y, median follow-up 15 months, range 1-32). The fovea was completely or partially detached in 16, attached in 12, and not assessable in 2 eyes on intraoperative imaging. Median BCVA at baseline was better in the pediatric group (p <0.05), and did not change significantly independent of age. Meaningful loss of BCVA (≥0.3 logMAR) occurred in 4/18 adult eyes, meaningful gain (≥-0.3 logMAR) in 2/18 adult and 2/8 pediatric eyes. LLVA and scotopic 2CTP improved considerably in pediatrics. Scotopic blue FST improved at all ages, more in pediatrics (8/8 eyes gain ≥ 10 dB, p<0.05). In pediatrics, median GVF improved by 20% for target V4e and by 50% for target III4e (target I4e not detected). Novel atrophy developed in 13/26 eyes at the site of the bleb and/or peripheral of vascular arcades. FST improvements did not correlate with development of chorioretinal atrophy at M12. Mean central retinal thickness was 166.7 μm (±25.45) at baseline (30 eyes) and 157.69 μm (±30.3) at M12 (26 eyes). Eight adult patients were treated unilaterally. The non-treated eyes did not show meaningful changes during follow-up.

CONCLUSIONS: These real-world data show effectiveness of VN therapy with stable median BCVA and mean retinal thickness, and improvements of LLVA, FST and 2CTP up to 32 months. Treatment effects were superior in the pediatric group. We observed new chorioretinal atrophy in 50% of the treated eyes.

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