Analysis of Data from Randomized Controlled Trials: A Practical Guide

✍ Scribed by Jos W.R. Twisk

• Publisher: Springer
• Year: 2021
• Tongue: English
• Leaves: 167
• Edition: 1st ed. 2021
• Category: Library

✦ Synopsis

This book provides a practical guide to the analysis of data from randomized controlled trials (RCT). It gives an answer to the question of how to estimate the intervention effect in an appropriate way. This problem is examined for different RCT designs, such as RCTs with one follow-up measurement, RCTs with more than one follow-up measurement, cluster RCTs, cross-over trials, stepped wedge trials, and N-of-1 trials. The statistical methods are explained in a non-mathematical way and are illustrated by extensive examples. All datasets used in the book are available for download, so readers can reanalyse the examples to gain a better understanding of the methods used. Although most examples are taken from epidemiological and clinical studies, this book is also highly recommended for researchers working in other fields.

✦ Table of Contents

Contents
Chapter 1: Introduction
1.1 Introduction
1.2 Intention-to-Treat Analysis
1.3 General Purpose and Prior Knowledge
1.4 Examples and Software
1.5 Equations
Chapter 2: Analysis of RCT Data with One Follow-Up Measurement
2.1 Statistical Methods
2.2 Example
Chapter 3: Analysis of RCT Data with More Than One Follow-Up Measurement
3.1 Introduction
3.2 Example
3.3 GLM for Repeated Measures
3.4 Regression-Based Methods
3.4.1 Longitudinal Analysis of Covariance
3.4.2 Repeated Measures
3.4.3 Analysis of Changes
3.5 Overview and Discussion
3.6 Recommendation
3.7 Should the Analysis Be Adjusted for Time?
3.8 Alternative Repeated Measures for the Analysis of an RCT with One Follow-Up Measurement
Chapter 4: Analysis of Data from a Cluster RCT
4.1 Introduction
4.2 Example with One Follow-Up Measurement
4.3 Example with More Than One Follow-Up Measurement
4.4 Comment
Chapter 5: Analysis of Data from a Cross-Over Trial
5.1 Introduction
5.2 Example
5.3 Alternative Analyses
Chapter 6: Analysis of Data from a Stepped Wedge Trial
6.1 Introduction
6.2 Example Dataset
6.3 Statistical Methods
6.3.1 Comparing Intervention and Control Measurements
6.3.2 Comparing Different Arms
6.3.3 Comparing Groups with a Different Number of Intervention Measurements
6.3.4 Comparing Transitions
6.4 A Second Example
6.4.1 Introduction
6.4.2 Comparing Intervention and Control Measurements
6.4.3 Comparing Different Arms
6.4.4 Comparing Groups with a Different Number of Intervention Measurements
6.4.5 Comparing Transitions
6.5 Comments
6.5.1 Adjustment for Time
6.5.2 Adjustment for the Baseline Value
6.5.3 Recommendation
Chapter 7: Analysis of Data from an N-of-1 Trial
7.1 Introduction
7.2 Example
Chapter 8: Dichotomous Outcomes
8.1 Introduction
8.2 RCT with a Dichotomous Outcome with One Follow-Up Measurement
8.3 RCT with a Dichotomous Outcome with More Than One Follow-Up Measurement
8.3.1 Example
8.4 Comments
8.4.1 Missing Data
8.4.2 Hypothesis Testing Versus Effect Estimation
8.4.3 Cluster RCT with a Dichotomous Outcome
8.5 The Problem of Non-Collapsibility
8.5.1 A Numerical Example
8.6 Other Outcomes
Chapter 9: What to Do When Only a Baseline Measurement Is Available
9.1 Introduction
9.2 Examples
9.2.1 RCT with One Follow-Up Measurement
9.2.2 RCT with More Than One Follow-Up Measurement
9.3 Comments
9.3.1 Sensitivity Analysis
9.3.2 Selective Imputation
9.3.3 Other Comments
9.4 Recommendation
Chapter 10: Sample Size Calculations
10.1 Introduction
10.2 Example
10.3 Comments
Chapter 11: Miscellaneous
11.1 Different Designs
11.2 Statistical Testing of Baseline Differences in an RCT
11.3 Analyzing Within-Group Changes in an RCT
11.3.1 Example
References
Index