Visual Acuity Change over 12 Months in the Prospective Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Study
Visual Acuity Change over 12 Months in the Prospective Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Study
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DOI：
10.1016/j.ophtha.2017.04.026
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发表时间：
2017-11-01
期刊：
影响因子：
13.7
通讯作者：
Scholl, Hendrik P. N.
中科院分区：
文献类型：
--
作者：
Kong, Xiangrong;Strauss, Rupert W.;Scholl, Hendrik P. N.
目的：评估最佳矫正视力（BCVA）的年变化率和1年内1线或1线以上丧失的风险，并确定Stargardt病（STGD1）患者BCVA丧失的危险因素。设计：多中心前瞻性队列研究。参与者：在美国和欧洲的9个中心招募了259名分子确诊的STGD1患者（489只眼睛）。方法：每6个月随访一次，分析基线和6、12个月随访时的数据。采用早期治疗糖尿病视网膜病变研究（ETDRS）方案测量最佳矫正视力。采用标准化报告表收集参与者的特征和临床观察结果。采用线性混合效应模型估计BCVA损失率。采用广义估计方程的线性模型来识别1年内1条或更多条BCVA损失的危险因素。主要观察指标：1年内BCVA的变化。结果：基线横断面分析显示，较早的症状出现和较长的发病时间与较差的BCVA相关。纵向分析显示12个月内BCVA总体无显著变化，但BCVA变化率与基线BCVA差异有统计学意义（P < 0.001）。眼睛的BCVA基线BCVA 20/25或更好的速度下降每年2.8 ETDRS字母(P = 0.10),眼睛之间的基线BCVA 20/25和20/70下降的速度每年2.3 ETDRS字母(P = 0.002),眼睛之间的基线BCVA 20/70和20/200下降的速度每年0.8 ETDRS字母(P = 0.08),和眼睛与基线BCVA比20/200显示每年2.3 ETDRS字母的显著改善(P < 0.001)。总体而言，12.9%的眼睛失去1条线或更多，这种BCVA丧失的风险因基线BCVA水平而异（P = 0.016）。吸烟和维生素A的使用与基线BCVA无显著相关，也与1年内BCVA损失率无显著相关。结论：STGD1患者BCVA在12个月期间的变化很小，但根据基线BCVA而变化。考虑到1年内的缓慢变化，BCVA不太可能是持续1年的STGD1治疗试验的敏感结果指标。(C) 2017年由美国眼科学会发布
Purpose: To estimate the yearly rate of change of best-corrected visual acuity (BCVA) and the risk of loss 1 line or more over 1 year and to identify risk factors for BCVA loss in patients with Stargardt disease (STGD1).Design: Multicenter, prospective cohort study.Participants: Two hundred fifty-nine patients (489 eyes) with molecularly confirmed STGD1 enrolled at 9 centers in the United States and Europe.Methods: Participants were followed up every 6 months, and data at the baseline and 6-and 12-month visits were analyzed. Best-corrected visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Standardized reporting forms were used to collect participants' characteristics and clinical observations. Linear mixed effects models were used to estimate the rate of BCVA loss. Linear models with generalized estimating equations were used to identify risk factors for BCVA loss of 1 line or more over 1 year.Main Outcome Measures: Change in BCVA over 1 year.Results: Cross-sectional analysis at baseline showed that earlier symptom onset and longer duration since onset was associated with worse BCVA. Longitudinal analysis showed no overall significant change of BCVA within 12 months, but the rate of BCVA change was significantly different by baseline BCVA (P < 0.001). The BCVA of eyes with baseline BCVA of 20/25 or better declined at a rate of 2.8 ETDRS letters per year (P = 0.10), eyes with baseline BCVA between 20/25 and 20/70 declined at a rate of 2.3 ETDRS letters per year (P = 0.002), eyes with baseline BCVA between 20/70 and 20/200 declined at a rate of 0.8 ETDRS letters per year (P = 0.08), and eyes with baseline BCVA worse than 20/200 showed a significant improvement of 2.3 ETDRS letters per year (P < 0.001). Overall, 12.9% of eyes lost 1 line or more, and the risk of such BCVA loss was different by baseline BCVA level (P = 0.016). Smoking and vitamin A use was not associated significantly with baseline BCVA, nor with rate of BCVA loss over 1 year.Conclusions: Change in BCVA in STGD1 patients over a 12-month period was small, but varied depending on baseline BCVA. Given the slow change during 1 year, BCVA is unlikely to be a sensitive outcome measure for STGD1 treatment trials with 1 year's duration. (C) 2017 by the American Academy of Ophthalmology