Novel vision tests: data analysis from a RP Lutein trial

描述(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，逐渐丧失周边视力，最终完全丧失视力。外科和内科的努力并不能减缓RP的光感受器退化。我们目前正在完成一项由NCCAM赞助的安慰剂对照交叉试验研究，该研究使用营养补充剂(叶黄素)，此前已被证明对RP患者的视力和中央视野面积有积极影响。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，其中包括几个最初未设想的指标，以及在每个参与者的家庭PC上进行每周自我管理的自动测试时收集的13个次要结果指标。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--一些是RP患者，一些是正常视力患者--收集数据以评估基于PC的测试的有效性。这为我们提供了一个独特的和非常大的结果数据集合，初步分析表明，基于PC的数据具有极好的重复性。叶黄素试点研究的范围有限，无法涵盖对这一大型数据收集的详细分析。我们建议通过分析它们的稳定性和相互一致性来验证基于PC的结果测量，并将它们与基于实验室的结果测量进行比较，在试验的前24周内，20-23名服用安慰剂补充剂的RP患者和没有服用叶黄素的受试者。此外，我们将使用补充叶黄素后基于实验室的主要和次要结果测量作为对基于PC的测试结果中观察到的任何变化的校准测量。对这些数据的详细研究不仅在评估叶黄素对RP患者视力的潜在影响和时间进程方面具有重要意义，而且在验证一系列经过良好校准的自动化家庭测试方面也具有重要意义，这些测试可能在未来的临床试验中提供一种廉价、有效和频繁的方法来监测两次临床就诊之间的视力。

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.