Bascom Palmer Eye Institute, Miami, Florida.
Bascom Palmer Eye Institute, Miami, Florida.
Ophthalmol Retina. 2022 Apr;6(4):273-283. doi: 10.1016/j.oret.2021.11.005. Epub 2021 Dec 9.
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.
Multicenter, retrospective, consecutive case series, and cross-sectional prospective survey.
All 41 consecutive patients treated with voretigene neparvovec-rzyl after Food and Drug Administration approval at 3 institutions between January 2018 and May 2020.
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.
Changes in best-corrected VA and retinal morphology and in patients' perceptions.
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.
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.
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。
多中心、回顾性、连续病例系列和横断面前瞻性调查。
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。
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。
最佳矫正视力和视网膜形态的变化以及患者的感知。
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。
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。
