Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland; Bloomberg School of Public Health, Johns Hopkins University, Baltimore, Maryland.
Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland; Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology, London, United Kingdom; Department of Ophthalmology, Medical University Graz and Johannes Kepler University Linz, Linz, Austria.
Ophthalmology. 2016 Sep;123(9):1887-97. doi: 10.1016/j.ophtha.2016.05.027. Epub 2016 Jul 2.
To examine the association between characteristics of Stargardt disease and visual acuity (VA), to estimate the longitudinal rate of VA loss, and to identify risk factors for VA loss.
Retrospective, multicenter cohort study.
A total of 176 patients (332 eyes) with molecularly and clinically confirmed Stargardt disease enrolled from the United States and Europe.
Standardized data report forms were used to collect retrospective data on participants' characteristics and best-corrected or presenting VA from medical charts. Linear models with generalized estimating equations were used to estimate the cross-sectional associations, and linear mixed effects models were used to estimate the longitudinal VA loss.
Yearly change in VA.
The median duration of observation was 3.6 years. At baseline, older age of symptom onset was associated with better VA, and a longer duration of symptoms was associated with worse VA. Longitudinal analysis estimated an average of 0.3 lines loss (P < 0.0001) per year overall, but the rate varied according to baseline VA: (1) eyes with baseline VA ≥20/25 (N = 53) declined at a rate of approximately 1.0 line per year; (2) eyes with VA between 20/25 and 20/70 (N = 65) declined at a rate of approximately 0.9 lines per year; (3) eyes with VA between 20/70 and 20/200 (N = 163) declined at a rate of 0.2 lines per year; and (4) eyes with VA worse than 20/200 (n = 49) improved at a rate of 0.5 lines per year. Older age of onset was associated with slower VA loss: Patients with onset age >30 years showed 0.4 lines slower change of VA per year (P = 0.01) compared with patients with onset age ≤14 years.
Given the overall slow rate of VA loss, VA is unlikely to be a sensitive outcome measure for treatment trials of Stargardt disease. However, given the faster decline in younger patients and those with no or mild visual impairment, VA may be a potential outcome measure for trials targeting such subgroups of patients. These observations will need to be assessed in a prospective study bearing in mind the inherent limitations of retrospective datasets.
研究斯塔加特病的特征与视力（VA）之间的关系，估计 VA 丧失的纵向速率，并确定 VA 丧失的危险因素。
回顾性、多中心队列研究。
共纳入来自美国和欧洲的 176 名（332 只眼）经分子和临床确诊的斯塔加特病患者。
使用标准化数据报告表从病历中收集参与者特征和最佳矫正或表现 VA 的回顾性数据。使用广义估计方程的线性模型来估计横断面相关性，使用线性混合效应模型来估计纵向 VA 丧失。
VA 的年变化。
中位观察时间为 3.6 年。在基线时，发病年龄较大与 VA 较好相关，而症状持续时间较长与 VA 较差相关。纵向分析估计总体上每年平均丧失 0.3 行（P < 0.0001），但速率根据基线 VA 而有所不同：（1）基线 VA ≥20/25（N = 53）的眼每年下降约 1.0 行；（2）VA 在 20/25 至 20/70 之间（N = 65）的眼每年下降约 0.9 行；（3）VA 在 20/70 至 20/200 之间（N = 163）的眼每年下降 0.2 行；（4）VA 差于 20/200（n = 49）的眼每年提高 0.5 行。发病年龄较大与 VA 丧失较慢相关：发病年龄 >30 岁的患者每年 VA 变化慢 0.4 行（P = 0.01），而发病年龄 ≤14 岁的患者每年 VA 变化快 0.4 行。
鉴于 VA 丧失的总体速度较慢，VA 不太可能成为斯塔加特病治疗试验的敏感结局指标。然而，鉴于年轻患者和无或轻度视力损害患者的 VA 下降较快，VA 可能是针对此类患者亚组的试验的潜在结局指标。这些观察结果需要在一项前瞻性研究中进行评估，同时考虑到回顾性数据集固有的局限性。
