The long-term natural history of geographic atrophy from age-related macular degeneration - Enlargement of atrophy and implications for interventional clinical trials
The long-term natural history of geographic atrophy from age-related macular degeneration - Enlargement of atrophy and implications for interventional clinical trials
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DOI：
10.1016/j.ophtha.2006.09.016
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发表时间：
2007-02-01
期刊：
影响因子：
13.7
通讯作者：
Bressler, Neil M.
中科院分区：
文献类型：
--
作者：
Sunness, Janet S.;Margalit, Eyal;Bressler, Neil M.
目的：报告地图样萎缩（GA）随时间的扩大率，其与基线萎缩大小和先前扩大率的关系，以及设计未来GA治疗试验的意义。设计：由年龄相关性黄斑变性引起的GA的前瞻性自然史研究。参与者：131例患者的212只眼纳入分析。每年随访包括立体彩色眼底照片。确定并测量GA的面积，并评估萎缩的扩大率。采用系统治疗和单眼治疗的临床试验的样本量被确定。主要结果测量：萎缩扩大率。结果：中位总体扩大率为2.1 mm（2）/年（平均2.6 mm（2）/年）。基线时萎缩面积较大的眼睛往往有较大的扩大率，但对先前扩大率的了解是预测随后扩大率的最重要因素。双侧GA患者的2只眼增大率之间具有高度一致性（相关系数，0.76）。为了检测系统治疗的增大率降低25%（α，0.05;把握度，0.80;失访，15%），对照组和治疗组各需要153例患者，每例患者随访2年。对于单眼治疗，38例患者与双边GA将是必需的，与未经治疗的眼睛作为对照治疗eye.Conclusions：治疗试验GA的结果变量的变化扩大率是可行的。
Purpose: To report the enlargement rate of geographic atrophy (GA) over time, its relationship to size of atrophy at baseline and to prior enlargement rate, and the implications for designing future treatment trials for GA.Design: Prospective natural history study of GA resulting from age-related macular degeneration.Participants: Two hundred twelve eyes of 131 patients were included in the analysis.Methods: Annual follow-up included stereo color fundus photographs. The areas of GA were identified and measured, and the rate of enlargement of the atrophy was assessed. Sample sizes for clinical trials using systemic treatment and uniocular treatment were determined.Main Outcome Measure: Rate of enlargement of the atrophy.Results: The median overall enlargement rate was 2.1 mm(2)/year (mean, 2.6 mm(2)/year). Eyes with larger areas of atrophy at baseline tended to have larger enlargement rates, but knowledge of prior rates of enlargement was the most significant factor in predicting subsequent enlargement rates. There was high concordance between the enlargement rates in the 2 eyes of patients with bilateral GA (correlation coefficient, 0.76). To detect a 25% reduction in enlargement rate for a systemic treatment (alpha, 0.05; power, 0.80; losses to follow-up, 15%), 153 patients each in a control and treatment group would be required for a trial with a 2-year follow-up period for each patient. For a uniocular treatment, 38 patients with bilateral GA would be required, with the untreated eye serving as a control for the treated eye.Conclusions: Treatment trials for GA with an outcome variable of change in enlargement rate are feasible.