The Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Studies Design and Baseline Characteristics: ProgStar Report No. 1
The Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Studies Design and Baseline Characteristics: ProgStar Report No. 1
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DOI：
10.1016/j.ophtha.2015.12.009
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发表时间：
2016-04-01
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：
Strauss, Rupert W.;Ho, Alex;Scholl, Hendrik P. N.
目的：描述Stargardt病（STGD 1）2项自然史研究患者的设计和基线特征。设计：多中心回顾性和前瞻性队列研究。参与者：来自美国和欧洲9个中心的365例基线年龄6岁及以上、携带ABCA 4基因致病变异体并伴有特定眼部病变的独特患者。方法：在回顾性研究中，患者提供了至少2次和最多4次访视的病历数据，至少有1种检查方式：眼底自发荧光（FAF）、光谱域（SD）光学相干断层扫描（SD OCT）和/或微视野检查（MP）。单次访视之间的总观察期至少为2年，最长为5年。还获得了人口统计学和视力（VA）数据。在前瞻性研究中，符合条件的患者进行了检查，在基线使用标准协议，6个月的后续访问计划为2年的时间内连续早期治疗糖尿病视网膜病变研究（ETDRS）最佳矫正VA，SD OCT，FAF，和MP.Main结局措施：设计和理由的多中心研究，以确定进展STGD 1在2个大型回顾性和前瞻性国际队列。两个队列的详细基线特征，包括人口统计学，结构和功能的视网膜metrics.Results：进入回顾性研究，251例患者（458只眼）入组;平均随访+/-标准差为3.9 +/- 1.6年。在基线时，36%的患者没有或有轻度视力丧失，47%的研究眼有明显的自体荧光减少（DAF），平均病变面积为2.5 ± 2.9 mm（2）（范围0.02-16.03 mm（2））。259例患者（489只眼）入组前瞻性研究。在基线时，20%的患者没有或有轻度VA损失，64%的患者有Doppler，平均病变面积为4.0 +/- 4.4 mm（2）（范围0.03-24.24 mm（2））。MP的平均视网膜敏感度为10.8 +/- 5.0 dB。结论：ProgStar队列的基线特征，包括广泛的疾病严重程度，并预计提供有价值的数据进展的基础上，来自多种方法的一系列定量测量，这将是至关重要的计划临床试验的设计。(C)2016年美国眼科学会。
Purpose: To describe the design and baseline characteristics of patients enrolled into 2 natural history studies of Stargardt disease (STGD1).Design: Multicenter retrospective and prospective cohort studies.Participants: Three hundred sixty-five unique patients aged 6 years and older at baseline harboring diseasecausing variants in the ABCA4 gene and with specified ocular lesions were enrolled from 9 centers in the United States and Europe.Methods: In the retrospective study, patients contributed medical record data from at least 2 and up to 4 visits for at least 1 examination modality: fundus autofluorescence (FAF), spectral-domain (SD) optical coherence tomography (SD OCT), and/or microperimetry (MP). The total observational period was at least 2 years and up to 5 years between single visits. Demographic and visual acuity (VA) data also were obtained. In the prospective study, eligible patients were examined at baseline using a standard protocol, with 6-month follow-up visits planned for a 2-year period for serial Early Treatment Diabetic Retinopathy Study (ETDRS) best-corrected VA, SD OCT, FAF, and MP.Main Outcome Measures: Design and rationale of a multicenter study to determine the progression of STGD1 in 2 large retrospective and prospective international cohorts. Detailed baseline characteristics of both cohorts are presented, including demographics, and structural and functional retinal metrics.Results: Into the retrospective study, 251 patients (458 eyes) were enrolled; mean follow-up +/- standard deviation was 3.9 +/- 1.6 years. At baseline, 36% had no or mild VA loss, and 47% of the study eyes had areas of definitely decreased autofluorescence (DDAF) with an average lesion area of 2.5 +/- 2.9 mm(2) (range, 0.02-16.03 mm(2)). Two hundred fifty-nine patients (489 eyes) were enrolled in the prospective study. At baseline, 20% had no or mild VA loss, and 64% had areas of DDAF with an average lesion area of 4.0 +/- 4.4 mm(2) (range, 0.03-24.24 mm(2)). The mean retinal sensitivity with MP was 10.8 +/- 5.0 dB.Conclusions: The ProgStar cohorts have baseline characteristics that encompass a wide range of disease severity and are expected to provide valuable data on progression based on serial quantitative measurements derived from multiple methods, which will be critical to the design of planned clinical trials. (C) 2016 by the American Academy of Ophthalmology.