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Medical Statistics from Scratch

An Introduction for Health Professionals

Second Edition

David Bowers

Honorary Lecturer, School of Medicine, University of Leeds, UK

Medical Statistics from Scratch

Second Edition

Medical Statistics from Scratch

An Introduction for Health Professionals

Second Edition

David Bowers

Honorary Lecturer, School of Medicine, University of Leeds, UK

Copyright C 2008 John Wiley & Sons Ltd, The Atrium, Southern Gate, Chichester,

West Sussex PO19 8SQ, England

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Library of Congress Cataloging-in-Publication Data

Bowers, David, 1938–

Medical statistics from scratch : an introduction for health professionals / David Bowers. — 2nd ed. p. ; cm.

Includes bibliographical references and index. ISBN 978-0-470-51301-9 (cloth : alk, paper)

1.Medical statistics. 2. Medicine—Research—Statistical methods. I. Title. [DNLM: 1. Biometry. 2. Statistics—methods. WA 950 B786m 2007] RA409.B669 2007

610.72’7—dc22

2007041619

British Library Cataloguing in Publication Data

A catalogue record for this book is available from the British Library

ISBN 978-0-470-51301-9

Typeset in 10/12pt Minion by Aptara Inc., New Delhi, India

Printed and bound in Great Britain by Antony Rowe Ltd., Chippenham, Wilts

This book is printed on acid-free paper responsibly manufactured from sustainable forestry in which at least two trees are planted for each one used for paper production.

This book is for Susanne

Contents

Preface to the 2nd Edition

xi

Preface to the 1st Edition

xiii

Introduction

xv

I Some Fundamental Stuff

1

1 First things first – the nature of data

3

Learning Objectives

3

Variables and data

3

The good, the bad, and the ugly – types of variable

4

Categorical variables

4

Metric variables

7

How can I tell what type of variable I am dealing with?

9

II Descriptive Statistics

15

2 Describing data with tables

17

Learning Objectives

17

What is descriptive statistics?

17

The frequency table

18

3 Describing data with charts

29

Learning Objectives

29

Picture it!

29

Charting nominal and ordinal data

30

Charting discrete metric data

34

Charting continuous metric data

35

Charting cumulative data

37

4 Describing data from its shape

43

Learning Objectives

43

The shape of things to come

43

viii CONTENTS

5 Describing data with numeric summary values

51

Learning Objectives

51

Numbers R us

52

Summary measures of location

54

Summary measures of spread

57

Standard deviation and the Normal distribution

65

III Getting the Data

69

6 Doing it right first time – designing a study

71

Learning Objectives

71

Hey ho! Hey ho! It’s off to work we go

72

Collecting the data – types of sample

74

Types of study

75

Confounding

81

Matching

81

Comparing cohort and case-control designs

83

Getting stuck in – experimental studies

83

IV From Little to Large – Statistical Inference

91

7 From samples to populations – making inferences

93

Learning Objectives

93

Statistical inference

93

8 Probability, risk and odds

97

Learning Objectives

97

Chance would be a fine thing – the idea of probability

98

Calculating probability

99

Probability and the Normal distribution

100

Risk

100

Odds

101

Why you can’t calculate risk in a case-control study

102

The link between probability and odds

103

The risk ratio

104

The odds ratio

105

Number needed to treat (NNT)

106

V The Informed Guess – Confidence Interval Estimation

109

9 Estimating the value of a single population parameter – the idea of

111

confidence intervals

Learning Objectives

111

Confidence interval estimation for a population mean

112

Confidence interval for a population proportion

116

Estimating a confidence interval for the median of a single population

117

CONTENTS

ix

10 Estimating the difference between two population parameters

119

Learning Objectives

119

What’s the difference?

120

Estimating the difference between the means of two independent populations – using

 

a method based on the two-sample t test

120

Estimating the difference between two matched population means – using a method

 

based on the matched-pairs t test

125

Estimating the difference between two independent population proportions

126

Estimating the difference between two independent population medians – the

 

Mann–Whitney rank-sums method

127

Estimating the difference between two matched population medians – Wilcoxon

 

signed-ranks method

131

11 Estimating the ratio of two population parameters

133

Learning Objectives

133

Estimating ratios of means, risks and odds

133

VI Putting it to the Test

139

12 Testing hypotheses about the difference between two

141

population parameters

Learning Objectives

141

The research question and the hypothesis test

142

A brief summary of a few of the commonest tests

144

Some examples of hypothesis tests from practice

146

Confidence intervals versus hypothesis testing

149

Nobody’s perfect – types of error

149

The power of a test

151

Maximising power – calculating sample size

152

Rules of thumb

152

13 Testing hypotheses about the ratio of two population parameters

155

Learning Objectives

155

Testing the risk ratio

155

Testing the odds ratio

158

14 Testing hypotheses about the equality of population proportions:

161

the chi-squared test

Learning Objectives

161

Of all the tests in all the world . . . the chi-squared (χ 2) test

162

VII Getting up Close

169

15 Measuring the association between two variables

171

Learning Objectives

171

Association

171

The correlation coefficient

175

x CONTENTS

16 Measuring agreement

181

Learning Objectives

181

To agree or not agree: that is the question

181

Cohen’s kappa

182

Measuring agreement with ordinal data – weighted kappa

184

Measuring the agreement between two metric continuous variables

184

VIII Getting into a Relationship

187

17 Straight line models: linear regression

189

Learning Objectives

189

Health warning!

190

Relationship and association

190

The linear regression model

192

Model building and variable selection

200

18 Curvy models: logistic regression

213

Learning Objectives

213

A second health warning!

213

Binary dependent variables

214

The logistic regression model

215

IX Two More Chapters

225

19 Measuring survival

227

Learning Objectives

227

Introduction

227

Calculating survival probabilities and the proportion surviving: the Kaplan-Meier table

228

The Kaplan-Meier chart

230

Determining median survival time

231

Comparing survival with two groups

232

20 Systematic review and meta-analysis

239

Learning Objectives

239

Introduction

240

Systematic review

240

Publication and other biases

244

The funnel plot

244

Combining the studies

246

Appendix: Table of random numbers

251

Solutions to Exercises

253

References

273

Index

277