Novel vision tests: data analysis from a RP Lutein trial

描述（由申请人提供）：视网膜色素变性（RP）在美国影响超过100，000人，并导致夜盲症，周边视力逐渐丧失，最终完全丧失视力。手术和医疗努力未能减缓RP中的感光细胞变性。我们目前正在完成一项NCCAM赞助的安慰剂对照的交叉试点研究，营养补充剂（叶黄素），这已被证明有一个积极的影响，视力和中央视野面积RP患者。ETDRS视力-每6周在实验室测量一次-是叶黄素研究的主要结果指标;在实验室访问期间收集了10个次要结果指标，包括最初没有设想的几个，以及在每个参与者的家庭PC上每周自我管理的自动测试期间收集的13个。 研究参与者包括43名服用叶黄素的RP患者，以及另外10名参与者-一些患有RP，一些视力正常-收集数据以允许基于PC的测试的有效性评估。这为我们提供了一个独特的和非常大的收集结果数据，初步分析表明，基于PC的具有良好的再现性。叶黄素试点研究的范围有限，无法涵盖对大量数据收集的详细分析。我们建议通过分析其稳定性和相互一致性来验证基于PC的结果测量，并将其与基于实验室的结果测量进行比较，在试验的前24周期间，对20-23名服用安慰剂补充剂的RP患者和未服用叶黄素的受试者进行比较。此外，我们将使用叶黄素补充后基于实验室的主要和次要结局指标作为基于PC的测试结果中观察到的任何变化的校准指标。这些数据的详细研究不仅在评估叶黄素对RP视力的潜在影响和时间过程中具有重要意义，而且在验证一系列校准良好的自动化家庭测试中也具有重要意义，这些测试可以提供一种廉价，有效和频繁的方法，用于在未来的临床试验中监测诊所访视之间的视力。

DESCRIPTION (provided by applicant): Retinitis pigmentosa (RP) affects over 100,000 individuals in the US, and causes nightblindness, gradual loss of peripheral vision, and eventually total vision loss. Surgical and medical efforts have not been able to slow down photoreceptor degeneration in RP. We are currently completing a NCCAM-sponsored placebo-controlled crossover pilot study with a nutrition supplement (lutein), which has previous been shown to have a positive effect on visual acuity and central visual field area in RP patients. ETDRS visual acuity-measured in the laboratory once per 6 weeks-is the primary outcome measure of the lutein study; 10 secondary outcome measures were collected during laboratory visits, including several not originally envisaged, and 13 more during weekly self-administered automated tests on each participant's home PC. Study participants included 43 RP patients taking lutein, and an additional 10 participants-some with RP, some normally-sighted-collecting data to allow validity assessment of the PC-based tests. This has provided us with a unique and very large collection of outcome data, and a preliminary analysis has shown that the PC-based have excellent reproducibility. The limited scope of the lutein pilot study could not cover a detailed analysis of this large data collection. We propose to validate PC-based outcome measures by analyzing their stability and mutual consistency, and by comparing them to lab-based outcome measures, for 20-23 RP patients on placebo supplements during the first 24 weeks of the trial and for subjects not taking lutein. In addition, we will use the lab-based primary and secondary outcome measures following lutein supplementation as calibration measures for any changes observed in the PC-based test outcomes. Detailed study of these data will not only be significant in assessing the potential effects and time course of lutein on vision in RP , but also in validating a collection of well-calibrated, automated home tests that may provide a cheap, effective, and frequent method for monitoring vision between clinic visits in future clinical trials.