The Vision Center, Department of Surgery, Children's Hospital Los Angeles, Los Angeles, California; Roski Eye Institute, Department of Ophthalmology, Keck School of Medicine, University of Southern California, Los Angeles, California.
Cincinnati Eye Institute, Cincinnati, Ohio; University of Cincinnati Department of Ophthalmology, Cincinnati, Ohio; Abrahamson Pediatric Eye Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Ophthalmol Retina. 2022 Jan;6(1):58-64. doi: 10.1016/j.oret.2021.03.016. Epub 2021 Apr 8.
To report an anatomic change following subretinal injection of voretigene neparvovec-rzyl (VN) for RPE65-mediated Leber congenital amaurosis.
Multicenter, retrospective chart review.
Patients who underwent subretinal VN injection at each of 4 participating institutions.
Patients were identified as having perifoveal chorioretinal atrophy if (1) the areas of atrophy were not directly related to the touch-down site of the subretinal cannula; and (2) the area of atrophy progressively enlarged over time. Demographic data, visual acuity, refractive error, fundus photographs, OCT, visual fields, and full-field stimulus threshold (FST) were analyzed.
Outcome measures included change in visual acuity, FST, visual fields, and location of atrophy relative to subretinal bleb position.
A total of 18 eyes of 10 patients who underwent subretinal injection of VN were identified as having developed perifoveal chorioretinal atrophy. Eight of 10 patients (80%) developed bilateral atrophy. The mean age was 11.6 years (range, 5-20 years), and 6 patients (60%) were male. Baseline mean logarithm of the minimum angle of resolution visual acuity and FST were 0.82 (standard deviation [SD], 0.51) and -1.3 log cd.s/m (SD, 0.44), respectively. The mean spherical equivalent was -5.7 diopters (D) (range, -11.50 to +1.75 D). Atrophy was identifiable at an average of 4.7 months (SD, 4.3) after surgery and progressively enlarged in all cases up to a mean follow-up period of 11.3 months (range, 4-18 months). Atrophy developed within and outside the area of the subretinal bleb in 10 eyes (55.5%), exclusively within the area of the bleb in 7 eyes (38.9%), and exclusively outside the bleb in 1 eye (5.5%). There was no significant change in visual acuity (P = 0.45). There was a consistent improvement in FST with a mean improvement of -3.21 log cd.s/m (P < 0.0001). Additionally, all 13 eyes with reliable Goldmann visual fields demonstrated improvement, but 3 eyes (23.1%) demonstrated paracentral scotomas related to the atrophy.
A subset of patients undergoing subretinal VN injection developed progressive perifoveal chorioretinal atrophy after surgery. Further study is necessary to determine what ocular, surgical delivery, and vector-related factors predispose to this complication.
报告雷柏西利替奈酚（VN）玻璃体内注射后出现的视网膜下注射相关解剖结构改变，该病例为 RPE65 相关莱伯先天性黑矇。
多中心、回顾性图表研究。
4 家参与机构中接受视网膜下 VN 注射的患者。
如果（1）萎缩区域与视网膜下套管的着陆点没有直接关系；（2）萎缩区域随时间逐渐扩大，则将患者识别为具有中心凹周围脉络膜视网膜萎缩。分析人口统计学数据、视力、屈光不正、眼底照片、OCT、视野和全视野刺激阈值（FST）。
观察指标包括视力、FST、视野和萎缩相对于视网膜下隆起位置的变化。
共确定了 18 只眼 10 名患者接受 VN 视网膜下注射后出现中心凹周围脉络膜视网膜萎缩。10 名患者中有 8 名（80%）出现双侧萎缩。平均年龄为 11.6 岁（范围 5-20 岁），6 名患者（60%）为男性。基线最小分辨角对数视力和 FST 的平均值分别为 0.82（标准差 [SD]，0.51）和-1.3 log cd·s/m（SD，0.44）。平均等效球镜度数为-5.7 屈光度（D）（范围-11.50 至+1.75 D）。术后平均 4.7 个月（SD，4.3）可识别萎缩，所有病例在平均 11.3 个月（范围 4-18 个月）的随访期间逐渐扩大。10 只眼（55.5%）的萎缩发生在视网膜下隆起区域内和周围，7 只眼（38.9%）的萎缩仅发生在隆起区域内，1 只眼（5.5%）的萎缩仅发生在隆起区域外。视力无显著变化（P=0.45）。FST 持续改善，平均改善-3.21 log cd·s/m（P<0.0001）。此外，所有 13 只具有可靠 Goldmann 视野的眼睛均有改善，但 3 只眼睛（23.1%）出现与萎缩相关的旁中心暗点。
接受视网膜下 VN 注射的患者中有一部分在手术后出现了进行性中心凹周围脉络膜视网膜萎缩。需要进一步研究以确定哪些眼部、手术输送和载体相关因素易导致这种并发症。
