Test-Retest Intervisit Variability of Functional and Structural Parameters in X-Linked Retinoschisis
Test-Retest Intervisit Variability of Functional and Structural Parameters in X-Linked Retinoschisis
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DOI：
10.1167/tvst.3.5.5
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发表时间：
2014-09-01
影响因子：
3
通讯作者：
Sieving, Paul A.
中科院分区：
文献类型：
--
作者：
Jeffrey, Brett G.;Cukras, Catherine A.;Sieving, Paul A.
目的：研究可用于解决x连锁青少年视网膜裂治疗试验安全性和有效性的四种结局指标的可变性。方法：对7名确诊RS1基因突变的男性进行为期6个月的4次就诊评估。评估包括视力、全视野视网膜电图（ERG）、显微黄斑灵敏度和光学相干断层扫描（OCT）测量的视网膜厚度。根据基线的视敏度将眼睛分为好眼组和差眼组。计算了每个参数的重复性系数，并使用叠刀重采样获得95%置信区间（ci）。结果：具有统计学意义的视力变化阈值范围为3到8个字母。对于ERG - a波，幅度减小大于56%被认为是显著的。对于其他参数，差眼组的可变性较低，可能是由于裂囊塌陷和/或视网膜萎缩造成的地板效应。三个重要参数的显著变化（更好/更差的眼睛）标准是：ERG b/a波比（0.44/0.23），点向灵敏度（10.4/7.0 dB）和视网膜中央厚度（31%/18%）。结论：本研究描述了x连锁青少年视网膜裂（XLRS）患者的视力、ERG、视网膜敏感性和相对于基线的视网膜中央厚度的95% CI范围。翻译相关性：定量了解结果测量的可变性对于建立XLRS患者治疗试验的安全性和有效性界限至关重要。
Purpose: To examine the variability of four outcome measures that could be used to address safety and efficacy in therapeutic trials with X-linked juvenile retinoschisis.Methods: Seven men with confirmed mutations in the RS1 gene were evaluated over four visits spanning 6 months. Assessments included visual acuity, full-field electroretinograms (ERG), microperimetric macular sensitivity, and retinal thickness measured by optical coherence tomography (OCT). Eyes were separated into Better or Worse Eye groups based on acuity at baseline. Repeatability coefficients were calculated for each parameter and jackknife resampling used to derive 95% confidence intervals (CIs).Results: The threshold for statistically significant change in visual acuity ranged from three to eight letters. For ERG a-wave, an amplitude reduction greater than 56% would be considered significant. For other parameters, variabilities were lower in the Worse Eye group, likely a result of floor effects due to collapse of the schisis pockets and/or retinal atrophy. The criteria for significant change (Better/Worse Eye) for three important parameters were: ERG b/a-wave ratio (0.44/0.23), point wise sensitivity (10.4/7.0 dB), and central retinal thickness (31%/18%).Conclusions: The 95% CI range for visual acuity, ERG, retinal sensitivity, and central retinal thickness relative to baseline are described for this cohort of participants with X-linked juvenile retinoschisis (XLRS).Translational Relevance: A quantitative understanding of the variability of outcome measures is vital to establishing the safety and efficacy limits for therapeutic trials of XLRS patients.