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A marginal rank-based inverse normal transformation approach to comparing multiple clinical trial endpoints

机译:基于边际等级的逆正态转化方法来比较多个临床试验终点

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摘要

The increase in incidence of obesity and chronic diseases and their health care costs have raised the importance of quality diet on the health policy agendas. The healthy eating index is an important measure for diet quality which consists of 12 components derived from ratios of dependent variables with distributions hard to specify, measurement errors and excessive zero observations difficult to model parametrically. Hypothesis testing involving data of such nature poses challenges because the widely used multiple comparison procedures such as Hotelling’s T2 test and Bonferroni correction may suffer from substantial loss of efficiency. We propose a marginal rank-based inverse normal transformation approach to normalizing the marginal distribution of the data before employing a multivariate test procedure. Extensive simulation was conducted to demonstrate the ability of the proposed approach to adequately control the type I error rate as well as increase the power of the test, with data particularly from non-symmetric or heavy-tailed distributions. The methods are exemplified with data from a dietary intervention study for type I diabetic children.
机译:肥胖症和慢性病的发病率及其医疗保健费用的增加,已使高质量饮食在卫生政策议程中的重要性提高。健康饮食指数是衡量饮食质量的重要指标,它由12个成分组成,这些成分来自因变量的比例,具有难以指定的分布,测量误差和难以通过参数建模的过多零观测值。涉及这种性质的数据的假设检验提出了挑战,因为广泛使用的多种比较程序(例如,Hotelling的T 2 检验和Bonferroni校正)可能会导致效率大幅下降。我们提出了一种基于边际秩的逆正态变换方法,以在采用多变量测试程序之前将数据的边际分布归一化。进行了广泛的仿真,以证明所提出的方法具有充分控制I型错误率以及提高测试能力的能力,尤其是来自非对称或重尾分布的数据。该方法以I型糖尿病儿童饮食干预研究的数据为例。

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