Statistical Methods for Ophthalmologic and Cluster Data

眼科和聚类数据的统计方法

• 批准号: 7260889
• 负责人: Bernard A Rosner
• 金额: $ 33.46万
• 依托单位: BRIGHAM AND WOMEN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 1998
• 资助国家: 美国
• 起止时间: 1998-08-01 至 2009-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7260889
• 关键词: Accounting; Area; Cataract; Data; Data Analyses; Diabetic Retinopathy; Disease; End Point; Event; Eye; Grant; Hypersensitivity; Individual; Internet; Methodology; Methods; Modeling; Nature; Ophthalmology; Outcome; Procedures; Randomized; Rank-Sum Tests; Research Personnel; SAS; Sample Size; Score; Site; Statistical Methods; Sum; Testing; Time; Visual Fields; Weight; comparison group; disorder subtype; programs; size

DESCRIPTION (provided by applicant): Ophthalmologic data present a data analyst with challenging methodologic problems. First, data are present for two eyes of an individual, which are usually positively correlated, but not the same. The challenge is how to use all the data in an efficient manner that accounts for the correlation between scores on fellow eyes. Substantial progress has been made in this area in the context of normally and binomially distributed outcome variables. SAS procedures PROC MIXED and PROC GENMOD deal effectively with these types of data. However, many scales in ophthalmology are ordinal in nature (e.g. ETDRS diabetic retinopathy scale, LOGS III cataract scale) and some continuous scales (e.g. Humphrey visual field) tend to be skewed and non-normally distributed. Hence, nonparametric analyses are desirable for two-group comparisons. In the previous cycle of this grant, we have extended the Wilcoxon rank sum test and signed rank test to account for clustered data in the setting where the unit of randomization is the subject, but the unit of analysis is the eye, and both eyes of a subject are randomized to the same treatment. In this renewal application, we extend these methods to cover the case when the unit of randomization is the subject, but fellow eyes may or may not receive the same treatment. This occurs commonly in ocular allergy trials. In addition, we propose to extend our methodology to provide estimates of power and sample size for clustered data analyzed by the Wilcoxon rank sum test, and assess efficiencies of different methods of weighting when clusters are of unequal size. Furthermore, we propose to apply our methodology for the Wilcoxon rank sum test for clustered data to the important problem of assessing goodness of fit of GEE models either when fit to longitudinal data or to cross-sectional data with correlated outcomes (e.g. fellow eyes). Finally, in the previous cycle of the grant we have studied methods for assessing whether exposure-disease relationships are the same for each component of a composite endpoint (e.g. different types of AMD), with the restriction that a subject can have only one type present. In this cycle, we propose to extend these methods to allow for the case where a subject can have multiple disease subtypes at the same time (e.g. different types of cataract). The methods developed in this proposal as well as in the previous cycle will be available in the form of SAS macros which can be accessed at the following web site URL:(http://www.qeocities.com/bernardrosner/channina.html).

描述（由申请方提供）：眼科数据给数据分析师带来了具有挑战性的方法学问题。首先，存在个体的两只眼睛的数据，其通常是正相关的，但不相同。挑战在于如何以有效的方式使用所有数据，以解释其他眼睛得分之间的相关性。在正态分布和二项分布结果变量的背景下，这一领域取得了重大进展。SAS程序PROC MIXED和PROC GENMOD有效地处理这些类型的数据。然而，眼科学中的许多量表在本质上是有序的（例如ETDRS糖尿病视网膜病变量表、LOGS III白内障量表），并且一些连续量表（例如Humphrey视野）倾向于偏斜和非正态分布。因此，两组比较需要非参数分析。在本基金的上一个周期中，我们扩展了Wilcoxon秩和检验和符号秩检验，以解释随机化单位为受试者，但分析单位为眼睛，受试者双眼随机接受相同治疗的情况下的聚类数据。在本更新申请中，我们将这些方法扩展到随机化单位为受试者但对侧眼可能接受或可能不接受相同治疗的情况。这通常发生在眼部过敏试验中。此外，我们建议扩展我们的方法，以提供估计的权力和样本量的聚类数据分析的Wilcoxon秩和检验，并评估不同的加权方法的效率时，集群的大小不等。此外，我们建议将我们的方法用于聚类数据的Wilcoxon秩和检验，以评估GEE模型拟合优度的重要问题，无论是在拟合纵向数据还是相关结果的横截面数据（例如，同胞的眼睛）。最后，在上一个资助周期中，我们研究了评估复合终点（例如不同类型的AMD）的每个组成部分的确定性-疾病关系是否相同的方法，限制是受试者只能有一种类型。在这个周期中，我们建议扩展这些方法，以允许受试者同时患有多种疾病亚型（例如不同类型的白内障）的情况。本提案以及前一周期中制定的方法将以SAS宏指令的形式提供，可在以下网址上查阅：（http：//www.qeocities.com/bernardrosner/channina.html）。