报告视网膜下注射voretigeneneparvovovec-rzyl(VN)治疗RPE65介导的Leber先天性黑蒙后的解剖学变化。
多中心,回顾性图表审查。
在4个参与机构中的每个机构接受视网膜下VN注射的患者。
如果(1)萎缩区域与视网膜下套管的触地部位不直接相关;和(2)萎缩区域随时间逐渐扩大,则患者被鉴定为患有中央凹脉络膜视网膜萎缩。人口统计数据,视敏度,屈光不正,眼底照片,OCT,视野,并分析了全场刺激阈值(FST)。
结果测量包括视敏度的变化,FST,视野,和萎缩的位置相对于视网膜下泡的位置。
10例接受视网膜下注射VN的患者的18只眼被确定为发生了中心凹周围脉络膜视网膜萎缩。10例患者中有8例(80%)出现双侧萎缩。平均年龄为11.6岁(范围,5-20年),6例(60%)为男性。分辨率视敏度和FST的最小角度的基线平均对数为0.82(标准偏差[SD],0.51)和-1.3logcd。s/m2(SD,0.44),分别。平均球面当量为-5.7屈光度(D)(范围,-11.50至+1.75D)。在平均4.7个月时可识别出萎缩(SD,4.3)手术后,在所有情况下逐渐扩大,平均随访期为11.3个月(范围,4-18个月)。10只眼(55.5%)在视网膜下泡区域内外出现萎缩,仅在7只眼睛的气泡区域内(38.9%),并且仅在1只眼睛的气泡外(5.5%)。视力无明显变化(P=0.45)。FST有一致的改善,平均改善为-3.21logcd。s/m2(P<0.0001)。此外,所有13只眼睛具有可靠的Goldmann视野表现出改善,但3眼(23.1%)显示与萎缩相关的中央旁暗点瘤。
接受视网膜下VN注射的患者亚组在手术后出现进行性中央凹脉络膜视网膜萎缩。进一步的研究是必要的,以确定哪些眼,手术分娩,和媒介相关因素导致这种并发症。
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/m2 (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/m2 (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.