Progression of Stargardt Disease as Determined by Fundus Autofluorescence Over a 12-Month Period: ProgStar Report No. 11
Progression of Stargardt Disease as Determined by Fundus Autofluorescence Over a 12-Month Period: ProgStar Report No. 11
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DOI：
10.1001/jamaophthalmol.2019.2885
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发表时间：
2019-10-01
影响因子：
8.1
通讯作者：
Smith, Monika
中科院分区：
文献类型：
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作者：
Strauss, Rupert W.;Kong, Xiangrong;Smith, Monika
重要性Stargardt病治疗试验需要对疾病进展进行敏感的结果测量。目的估计Stargardt病继发性萎缩进展的前瞻性自然史中萎缩性病变的进展率（ProgStar）研究，为期12个月。这项多中心前瞻性队列研究于2013年10月21日在国际选择的三级转诊中心进行，到2017年2月15日受Stargardt病影响的患者，基线时年龄≥ 6岁，并且携带ABCA 4基因的致病变体，在美国，英国和欧洲大陆的9个中心入组。数据分析发生在2016年11月至2017年1月。EXPOSURES将使用标准方案获得的自体荧光图像发送到中央阅读中心，并概述和定量确定自体荧光减少的区域、可疑自体荧光减少的区域以及自体荧光减少的总组合区域。以时间为自变量，采用线性混合模型估计进展率。主要结果和指标采用自体荧光成像测量萎缩性病变的生长，评估年进展率。（488只眼睛; 230名个体[88.8%]接受双眼检查）入组（首次访视时的平均[SD]年龄，33.3 [15.1]岁; 118名[54.4%]女性）。基线时480只眼和12个月后454只眼的分级图像可用于评价。基线时，306只眼睛的自体荧光明显减少，平均（SD）病变大小为3.93（4.37）mm（2）。基线时自发荧光减少的平均总面积为4.07（4.04）mm（2）。自体荧光明显减少的估计进展为每年0.76（95%CI，0.54-0.97）mm（2）（P
IMPORTANCE Sensitive outcome measures for disease progression are needed for treatment trials of Stargardt disease.OBJECTIVE To estimate the progression rate of atrophic lesions in the prospective Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) study over a 12-month period.DESIGN, SETTING, AND PARTICIPANTS This multicenter prospective cohort study was conducted in an international selection of tertiary referral centers from October 21, 2013, to February 15, 2017. Patients who were affected by Stargardt disease, aged 6 years and older at baseline, and harboring disease-causing variants of the ABCA4 gene were enrolled at 9 centers in the United States, United Kingdom, and continental Europe. Data analysis occurred from November 2016 to January 2017.EXPOSURES Autofluorescence images obtained with a standard protocol were sent to a central reading center, and areas of definitely decreased autofluorescence, questionably decreased autofluorescence, and the total combined area of decreased autofluorescence were outlined and quantified. Progression rates were estimated from linear mixed models with time as the independent variable.MAIN OUTCOMES AND MEASURES Yearly rate of progression, using the growth of atrophic lesions measured by autofluorescence imaging.RESULTS A total of 259 study participants (488 eyes; 230 individuals [88.8%] were examined in both eyes) were enrolled (mean [SD] age at first visit, 33.3 [15.1] years; 118 [54.4%] female). Gradable images were available for evaluation for 480 eyes at baseline and 454 eyes after 12 months. At baseline, definitely decreased autofluorescence was present in 306 eyes, and the mean (SD) lesion size was 3.93 (4.37) mm(2). The mean total area of decreased autofluorescence at baseline was 4.07 (4.04) mm(2). The estimated progression of definitely decreased autofluorescence was 0.76 (95% CI, 0.54-0.97) mm(2) per year (P