Differential Disease Progression in Atrophic Age-Related Macular Degeneration and Late-Onset Stargardt Disease
Differential Disease Progression in Atrophic Age-Related Macular Degeneration and Late-Onset Stargardt Disease
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DOI：
10.1167/iovs.16-20980
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发表时间：
2017-02-01
影响因子：
4.4
通讯作者：
Fleckenstein, Monika
中科院分区：
文献类型：
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作者：
Lindner, Moritz;Lambertus, Stanley;Fleckenstein, Monika
目的.比较年龄相关性黄斑变性（AMD）和迟发性Stargardt病（STGD 1）继发视网膜色素上皮（RPE）萎缩的病程。通过眼底自发荧光、近红外反射成像和最佳矫正视力（BCVA）对患者进行纵向检查。使用半自动化软件定量RPE萎缩的区域，并基于自体荧光和近红外反射图像评估中央凹的状态。采用混合效应模型比较萎缩进展率。BCVA损失和中央凹完整性的损失进行了分析，使用特恩布尔的估计。共有151例（226只眼）继发于AMD的RPE萎缩患者和38例（66只眼）继发于晚发型STGD 1的RPE萎缩患者接受了检查，中位时间为2.3年（四分位距，2.7）。AMD患者的平均基线年龄为74.2岁（SD，7.6），晚发型STGD 1患者为63.4岁（SD，9.9）（P = 1.1 × 10 - 7）。与晚发型STGD 1相比，AMD的平方根萎缩进展明显更快（0.28 mm/年[SE，0.01] vs. 0.23 [SE，0.03]; P = 0.030）。在晚发型STGD 1中，与AMD患者相比，中心凹的中位生存期显著延长（8.60 vs. 3.35年; P = 0.005），BCVA有晚3行的趋势（5.97 vs. 4.37年; P = 0.382）。这些自然史数据表明AMD与晚发型STGD 1的疾病进展不同。这些结果强调了在干预性试验的预后和设计方面，存在RPE萎缩的老年患者的精细表型的相关性。
PURPOSE. To compare the disease course of retinal pigment epithelium (RPE) atrophy secondary to age-related macula degeneratio (AMD) and late-onset Stargardt disease (STGD1).METHODS. Patients were examined longitudinally by fundus autofluorescence, near-infrared reflectance imaging, and best-corrected visual acuity (BCVA). Areas of RPE atrophy were quantified using semi-automated software, and the status of the fovea was evaluated based on autofluorescence and near-infrared reflectance images. Mixed-effects models were used to compare atrophy progression rates. BCVA loss and loss of foveal integrity were analyzed using Turnbull's estimator.RESULTS. A total of 151 patients (226 eyes) with RPE atrophy secondary to AMD and 38 patients (66 eyes) with RPE atrophy secondary to late-onset STGD1 were examined for a median time of 2.3 years (interquartile range, 2.7). Mean baseline age was 74.2 years (SD, 7.6) in AMD and 63.4 (SD, 9.9) in late-onset STGD1 (P = 1.1 X 10(-7)). Square root atrophy progression was significantly faster in AMD when compared with late-onset STGD1 (0.28 mm/year [SE, 0.01] vs. 0.23 [SE, 0.03]; P = 0.030). In late-onset STGD1, the median survival of the fovea was significantly longer when compared with eyes with AMD (8.60 vs. 3.35 years; P = 0.005) with a trend to a later BCVA loss of >= 3 lines (5.97 vs. 4.37 years; P = 0.382).CONCLUSIONS. These natural history data indicate differential disease progression in AMD versus late-onset STGD1. The results underline the relevance of refined phenotyping in elderly patients presenting with RPE atrophy in regard to prognosis and design of interventional trials.