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视觉敏锐度、视网膜形态和 RPE65 相关视网膜疾病接受 Voretigene Neparovec-rzyl 治疗后的患者感知。

Visual Acuity, Retinal Morphology, and Patients' Perceptions after Voretigene Neparovec-rzyl Therapy for RPE65-Associated Retinal Disease.

机构信息

Bascom Palmer Eye Institute, Miami, Florida.

Bascom Palmer Eye Institute, Miami, Florida.

出版信息

Ophthalmol Retina. 2022 Apr;6(4):273-283. doi: 10.1016/j.oret.2021.11.005. Epub 2021 Dec 9.

Abstract

OBJECTIVE

To explore the effect of patients' age, baseline visual acuity (VA), and intraoperative foveal detachment on outcomes of subretinal voretigene neparvovec-rzyl (Luxturna) therapy and to assess patients' perceptions of the treatment effect.

DESIGN

Multicenter, retrospective, consecutive case series, and cross-sectional prospective survey.

PARTICIPANTS

All 41 consecutive patients treated with voretigene neparvovec-rzyl after Food and Drug Administration approval at 3 institutions between January 2018 and May 2020.

METHODS

A retrospective chart review of operative reports, clinical notes, ancillary testing, and complications, comparing data at baseline and at 1, 2 to 3, 6 to 9, and 10 to 15 months after subretinal surgery was conducted. A survey was administered to adult patients and parents of pediatric patients.

MAIN OUTCOME MEASURES

Changes in best-corrected VA and retinal morphology and in patients' perceptions.

RESULTS

Seventy-seven eyes of 41 patients (16 adults and 25 pediatric patients; age range, 2-44 years; mean follow-up, 10 months [range, 1 week to 18.5 months]) were analyzed. There was no statistically significant vision change for the adults, whereas there was a trend of improvement for pediatric patients, which reached statistical significance for some time points. The baseline VA did not affect the posttherapy VA (P = 0.23). The central foveal thickness decreased mildly in both pediatric patients and adults, without significant differences between the populations. The fovea was detached by voretigene neparvovec-rzyl in 62 (81%) eyes. The inner segment-outer segment junction remained unchanged in 91% of 54 eyes with gradable OCT, with or without foveal detachment. Thirty-two (78%) patients were reached for the survey an average of 1.15 ± 0.50 years (range, 0.31 to 2.31) after the surgery in the first eye. Improvement in night, day, or color vision was reported by 23 (72%), 22 (69%), and 18 (56%) patients, respectively.

CONCLUSIONS

This study is limited by the large variability in follow-up time. There were no persistent statistically significant vision changes. A decrease in foveal thickness was noted in most eyes, but the long-term significance of this remains to be determined.

摘要

目的

探讨患者年龄、基线视力(VA)和术中黄斑脱离对视网膜下注射维替泊芬替加奈肽(Luxturna)治疗结果的影响,并评估患者对治疗效果的感知。

设计

多中心、回顾性、连续病例系列和横断面前瞻性调查。

参与者

2018 年 1 月至 2020 年 5 月,3 家机构在食品和药物管理局批准后,连续 41 例接受维替泊芬替加奈肽治疗的患者。

方法

对手术报告、临床记录、辅助检查和并发症进行回顾性图表审查,比较基线和术后 1、2 至 3、6 至 9 和 10 至 15 个月时的数据。对成年患者和儿科患者的父母进行了一项调查。

主要观察指标

最佳矫正视力和视网膜形态的变化以及患者的感知。

结果

对 41 例患者(16 例成年患者和 25 例儿科患者;年龄范围为 2-44 岁;平均随访时间为 10 个月[范围为 1 周至 18.5 个月])的 77 只眼进行了分析。成年患者的视力没有统计学上的显著变化,而儿科患者的视力有改善的趋势,在一些时间点达到了统计学意义。基线 VA 不影响治疗后的 VA(P=0.23)。黄斑中心凹厚度在儿科患者和成人中均轻度下降,但人群之间无显著差异。维替泊芬替加奈肽可使 62 只(81%)眼的黄斑脱离。在 54 只可分级 OCT 眼的内节-外节连接中,无论是否有黄斑脱离,91%保持不变。在第一只眼手术后平均 1.15±0.50 年(范围为 0.31 至 2.31)时,对 32 例(78%)患者进行了调查。23 例(72%)、22 例(69%)和 18 例(56%)患者分别报告夜间、白天或色觉改善。

结论

本研究受到随访时间差异较大的限制。没有持续的统计学显著视力变化。大多数眼的黄斑厚度下降,但长期意义尚待确定。

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