Analysis of data from randomized controlled trials: a practical guide
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| Format: | Book |
| Language: | English |
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Cham, Switzerland
Springer
[2021]
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| Subjects: | |
| Online Access: | Inhaltsverzeichnis Klappentext |
| Physical Description: | ix, 168 Seiten Illustrationen, Diagramme |
| ISBN: | 9783030818678 |
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| adam_text | Contents 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.............................................................................................. 1 1 2 3 3 3 2 Analysis of RCT Data with One Follow-Up Measurement................. 2.1 Statistical Methods.............................................................................. 2.2 Example................................................................................................ 5 5 10 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.................................................. 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.............................................................................................. 15 15 16 16 19 21 27 34 39 44 45 45 49 49 51 53 57 vii
viii Contents 5 Analysis of Data from a Cross-Over Trial............................................. 5.1 Introduction......................................................................................... 5.2 Example............................................................................................... 5.3 Alternative Analyses.......................................................................... 61 61 63 68 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................................................................. 73 73 75 75 75 79 98 100 102 103 104 104 7 Analysis of Data from an A-of-l Trial.................................................... 7.1 Introduction......................................................................................... 7.2 Exampie............................................................................................... 107 107 108 8 Dichotomous Outcomes.............................................................................. 8.1 Introduction......................................................................................... 8.2 RCT with a Dichotomous Outcome withOne Follow-Up Measurement...................................................................................... 8.3 RCT with a Dichotomous Outcome withMore 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.................................................................................. 115 115 84 87 91 91 92 94 116 118 119 126 128 130 130 132 133 134
Contents ix 9 What to Do When Onlya Baseline Measurement Is Available.... 137 9.1 Introduction......................................................................................... 137 9.2 Examples............................................................................................. 138 9.2.1 RCT with One Follow-UpMeasurement........................... 138 9.2.2 RCT with More Than One Follow-UpMeasurement. . . 142 9.3 Comments............................................................................................ 148 9.3.1 Sensitivity Analysis.............................................................. 148 9.3.2 Selective Imputation............................................................. 148 9.3.3 Other Comments.................................................................. 149 9.4 Recommendation................................................................................. 150 10 Sample Size Calculations........................................................................... 10.1 Introduction.......................................................................................... 10.2 Example............................................................................................... 10.3 Comments............................................................................................ 151 151 154 155 11 Miscellaneous............................................................................................... 11.1 Different Designs................................................................................ 11.2 Statistical Testing of
Baseline Differences inan RCT..................... 11.3 Analyzing Within-Group Changes in anRCT................................. 11.3.1 Example................................................................................ 157 157 158 158 159 References.............................................................................................................. 163 Index....................................................................................................................... 167
Jos W. R. Twisk Analysis of Data from Randomized Controlled Trials A Practical Guide 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-i 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.
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| adam_txt |
Contents 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. 1 1 2 3 3 3 2 Analysis of RCT Data with One Follow-Up Measurement. 2.1 Statistical Methods. 2.2 Example. 5 5 10 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. 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. 15 15 16 16 19 21 27 34 39 44 45 45 49 49 51 53 57 vii
viii Contents 5 Analysis of Data from a Cross-Over Trial. 5.1 Introduction. 5.2 Example. 5.3 Alternative Analyses. 61 61 63 68 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. 73 73 75 75 75 79 98 100 102 103 104 104 7 Analysis of Data from an A-of-l Trial. 7.1 Introduction. 7.2 Exampie. 107 107 108 8 Dichotomous Outcomes. 8.1 Introduction. 8.2 RCT with a Dichotomous Outcome withOne Follow-Up Measurement. 8.3 RCT with a Dichotomous Outcome withMore 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. 115 115 84 87 91 91 92 94 116 118 119 126 128 130 130 132 133 134
Contents ix 9 What to Do When Onlya Baseline Measurement Is Available. 137 9.1 Introduction. 137 9.2 Examples. 138 9.2.1 RCT with One Follow-UpMeasurement. 138 9.2.2 RCT with More Than One Follow-UpMeasurement. . . 142 9.3 Comments. 148 9.3.1 Sensitivity Analysis. 148 9.3.2 Selective Imputation. 148 9.3.3 Other Comments. 149 9.4 Recommendation. 150 10 Sample Size Calculations. 10.1 Introduction. 10.2 Example. 10.3 Comments. 151 151 154 155 11 Miscellaneous. 11.1 Different Designs. 11.2 Statistical Testing of
Baseline Differences inan RCT. 11.3 Analyzing Within-Group Changes in anRCT. 11.3.1 Example. 157 157 158 158 159 References. 163 Index. 167
Jos W. R. Twisk Analysis of Data from Randomized Controlled Trials A Practical Guide 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-i 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. |
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| spelling | Twisk, Jos W. R. 1962- Verfasser (DE-588)173605362 aut Analysis of data from randomized controlled trials a practical guide Jos W.R. Twisk Cham, Switzerland Springer [2021] ix, 168 Seiten Illustrationen, Diagramme txt rdacontent n rdamedia nc rdacarrier Statistical Theory and Methods Biomedicine, general Biostatistics Statistics Medicine Datenanalyse (DE-588)4123037-1 gnd rswk-swf Kontrollierte klinische Studie (DE-588)4202997-1 gnd rswk-swf Kontrollierte klinische Studie (DE-588)4202997-1 s Datenanalyse (DE-588)4123037-1 s DE-604 Erscheint auch als Online-Ausgabe 978-3-030-81865-4 Digitalisierung UB Augsburg - ADAM Catalogue Enrichment application/pdf http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=033914970&sequence=000001&line_number=0001&func_code=DB_RECORDS&service_type=MEDIA Inhaltsverzeichnis Digitalisierung UB Augsburg - ADAM Catalogue Enrichment application/pdf http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=033914970&sequence=000003&line_number=0002&func_code=DB_RECORDS&service_type=MEDIA Klappentext |
| spellingShingle | Twisk, Jos W. R. 1962- Analysis of data from randomized controlled trials a practical guide Statistical Theory and Methods Biomedicine, general Biostatistics Statistics Medicine Datenanalyse (DE-588)4123037-1 gnd Kontrollierte klinische Studie (DE-588)4202997-1 gnd |
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| title | Analysis of data from randomized controlled trials a practical guide |
| title_auth | Analysis of data from randomized controlled trials a practical guide |
| title_exact_search | Analysis of data from randomized controlled trials a practical guide |
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| title_full | Analysis of data from randomized controlled trials a practical guide Jos W.R. Twisk |
| title_fullStr | Analysis of data from randomized controlled trials a practical guide Jos W.R. Twisk |
| title_full_unstemmed | Analysis of data from randomized controlled trials a practical guide Jos W.R. Twisk |
| title_short | Analysis of data from randomized controlled trials |
| title_sort | analysis of data from randomized controlled trials a practical guide |
| title_sub | a practical guide |
| topic | Statistical Theory and Methods Biomedicine, general Biostatistics Statistics Medicine Datenanalyse (DE-588)4123037-1 gnd Kontrollierte klinische Studie (DE-588)4202997-1 gnd |
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