Design and analysis of quality of life studies in clinical trials : interdisciplinary statistics
Design and analysis of quality of life studies in clinical trials : interdisciplinary statistics
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临床试验中生活质量研究的设计和分析：跨学科统计
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发表时间：
2002
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通讯作者：
D. Fairclough
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作者：
D. Fairclough
实施例1：辅助乳腺癌试验实施例2：晚期非小细胞肺癌（NSCLC）实施例3：肾细胞癌试验概要研究设计和方案制定引言背景和原理研究目的受试者选择纵向设计生活质量测量指标选择进行纵向研究的概要模型引言构建分析模型构建重复测量模型构建生长曲线模型总结缺失数据介绍缺失数据模式缺失数据机制总结可忽略缺失数据的分析方法重复单变量分析多变量方法基线评估作为协变量较基线的变化经验贝叶斯估计值总结SIMPLE IMPUTATION介绍均值替代显式回归模型末值结转低估方差敏感性分析总结多重插补介绍多重插补概述显式单变量回归最近邻和预测均值匹配非单调缺失数据的近似贝叶斯Bootstrap多变量程序结合M分析敏感性分析插补与分析模型对设计的影响总结模式混合模型介绍双变量数据（两次重复测量）单调脱落参数模型附加阅读代数详细信息总结随机效应混合，共享参数，与选择模型介绍条件线性模型单调脱落的效果和脱落时间选择模型高级阅读摘要摘要测量方法引言选择摘要测量方法构建摘要测量方法不同时间的汇总统计量不同HRQoL领域或子量表的汇总高级注释总结多个终点介绍背景概念和定义多变量统计单变量统计恢复技术总结设计：分析计划介绍纵向数据的一般分析计划模型终点的多重性样本量和功效报告的结果总结附录书目
INTRODUCTION Health-Related Quality of Life Measuring Health-Related Quality of Life Example 1: Adjuvant Breast Cancer Trial Example 2: Advanced Non-Small-Cell Lung Cancer (NSCLC) Example 3: Renal Cell Carcinoma Trial Summary STUDY DESIGN AND PROTOCOL DEVELOPMENT Introduction Background and Rationale Research Objectives Selection of Subjects Longitudinal Designs Selection of a Quality of Life Measure Conduct Summary MODELS FOR LONGITUDINAL STUDIES Introduction Building the Analytic Models Building Repeated Measures Models Building Growth Curve Models Summary MISSING DATA Introduction Patterns of Missing Data Mechanisms of Missing Data Summary ANALYTIC METHODS FOR IGNORABLE MISSING DATA Introduction Repeated Univariate Analyses Multivariate Methods Baseline Assessment as a Covariate Change from Baseline Empirical Bayes Estimates Summary SIMPLE IMPUTATION Introduction Mean Value Substitution Explicit Regression Models Last Value Carried Forward Underestimation of Variance Sensitivity Analysis Summary MULTIPLE IMPUTATION Introduction Overview of Multiple Imputation Explicit Univariate Regression Closest Neighbor and Predictive Mean Matching Approximate Bayesian Bootstrap Multivariate Procedures for Nonmonotone Missing Data Combining the M Analyses Sensitivity Analyses Imputation vs. Analytic Models Implications for Design Summary PATTERN MIXTURE MODELS Introduction Bivariate Data (Two Repeated Measures) Monotone Dropout Parametric Models Additional Reading Algebraic Details Summary RANDOM-EFFECTS MIXTURE, SHARED-PARAMETER, AND SELECTION MODELS Introduction Conditional Linear Model Joint Mixed-Effects and Time to Dropout Selection Model for Monotone Dropout Advanced Readings Summary SUMMARY MEASURES Introduction Choosing a Summary Measure Constructing Summary Measures Summary Statistics across Time Summarizing Across HRQoL Domains or Subscales Advanced Notes Summary MULTIPLE ENDPOINTS Introduction Background Concepts and Definitions Multivariate Statistics Univariate Statistics Resampling Techniques Summary DESIGN: ANALYSIS PLANS Introduction General Analysis Plan Models for Longitudinal Data Multiplicity of Endpoints Sample Size and Power Reported Results Summary APPENDICES BIBLIOGRAPHY