Medical Statistics

Medical Statistics
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Описание книги

The 5th edition of this popular introduction to statistics for the medical and health sciences has undergone a significant revision, with several new chapters added and examples refreshed throughout the book. Yet it retains its central philosophy to explain medical statistics with as little technical detail as possible, making it accessible to a wide audience.  Helpful multi-choice exercises are included at the end of each chapter, with answers provided at the end of the book. Each analysis technique is carefully explained and the mathematics kept to minimum. Written in a style suitable for statisticians and clinicians alike, this edition features many real and original examples, taken from the authors' combined many years' experience of designing and analysing clinical trials and teaching statistics. Students of the health sciences, such as medicine, nursing, dentistry, physiotherapy, occupational therapy, and radiography should find the book useful, with examples relevant to their disciplines. The aim of training courses in medical statistics pertinent to these areas is not to turn the students into medical statisticians but rather to help them interpret the published scientific literature and appreciate how to design studies and analyse data arising from their own projects. However, the reader who is about to design their own study and collect, analyse and report on their own data will benefit from a clearly written book on the subject which provides practical guidance to such issues. The practical guidance provided by this book will be of use to professionals working in and/or managing clinical trials, in academic, public health, government and industry settings, particularly medical statisticians, clinicians, trial co-ordinators. Its practical approach will appeal to applied statisticians and biomedical researchers, in particular those in the biopharmaceutical industry, medical and public health organisations.

Оглавление

David Machin. Medical Statistics

Table of Contents

List of Tables

List of Illustrations

Guide

Pages

Medical Statistics. A Textbook for the Health Sciences

Preface

1 Uses and Abuses of Medical Statistics

Summary

1.1 Introduction

1.2 Why Use Statistics?

1.3 Statistics is About Common Sense and Good Design

Example from the Literature – Drinking Coffee and Cancer (IARC 2018)

1.4 How a Statistician Can Help

Sample Size and Power Considerations

Questionnaires

Choice of Sample and of Control Subjects

Design of Study

Laboratory Experiments

Displaying Data

Choice of Summary Statistics and Statistical Analysis

Medical Statistics and Data Science

2 Displaying and Summarising Data

Summary

2.1 Types of Data

Example from the Literature – Salicylic Acid Plasters for Treatment of Foot Corns

Categorical or Qualitative Data. Nominal Categorical Data

Ordinal Data

Ranks

Numerical or Quantitative Data. Count Data

Measured or Numerical Continuous

Interval and Ratio Scales

2.2 Summarising Categorical Data

Illustrative Example – Salicylic Acid Plasters for Treatment of Foot Corns

Labelling Binary Outcomes

2.3 Displaying Categorical Data

2.4 Summarising Continuous Data

Measures of Location – The Three ‘Ms’ – Mean, Median and Mode. Mean or Average

Example – Calculation of the Mean – Corn Size Data (mm)

Median

Example – Calculation of the Median – Corn Size Data

Mode

Example – Calculation of the Mode – Corn Size Data

Measures of Dispersion or Variability

Range

Example – Calculation of the Range – Corn Size Data

Quartiles and the Interquartile Range

Percentiles

Example – Calculation of the Range, Quartiles, and Inter‐Quartile Range – Corn Size Data

Standard Deviation and Variance

Illustrative Example – Calculation of the Standard Deviation – Foot Corn Size

Why is the Standard Deviation Useful?

Means or Medians?

2.5 Displaying Continuous Data

Dot Plots

Example – Dot Plot – Baseline Corn Size

Histograms

Box and Whisker Plot

Illustrative Example – Box and Whisker Plot – Birthweight by Type of Delivery

Scatter Plots

Illustrative Example – Scatter Plot – Baseline Corn Size by Corn Size at a Three month Follow‐up

Measures of Symmetry

2.6 Within‐Subject Variability

Illustrative Example – Within‐Subject Variability – Total Steps per Day

2.7 Presentation. Graphs

Tables

2.8 Points When Reading the Literature

2.9 Technical Details. Calculating the Sample Median

Calculating the Quartiles and Inter Quartile Range

2.10 Exercises

3 Summary Measures for Binary Data

Summary

3.1 Summarising Binary and Categorical Data

Ratios, Proportions, Percentages, Risk and Rates

Illustrative Example – RCT of Salicylic Acid Plasters for Treatment of Foot Corns

Labelling Binary Outcomes

Comparing Outcomes for Binary Data

Summarising Comparative Binary Data – Differences in Proportions

Example – Summarising Results from a Clinical Trial – Corn Plasters RCT: Differences in Proportions

Summarising Comparative Binary Data – Relative Risk

Example – Summarising Results from a Clinical Trial – Corn Plasters RCT: Relative Risk

Summarising Comparative Binary Data – Number Need to Treat

Example – Summarising Results from a Clinical Trial – Corn Plasters RCT: NNT

Issues with NNT – Always Consider all the Risks

Example – Importance of Considering both Absolute Risk and Relative Risk

Summarising Binary Data – Odds and Odds Ratios

Example – Summarising Results from a Clinical Trial– Corn Plasters: Odds Ratio

Why Should One Use the Odds Ratio?

The Odd Ratios are Symmetrical but the Relative Risk Is Not

How Are Risks Compared?

3.2 Points When Reading the Literature

3.3 Exercises

4 Probability and Distributions

Summary

4.1 Types of Probability

Examples of Addition and Multiplication Rules – Using Dice Rolling

Probability Distributions for Discrete Outcomes

4.2 The Binomial Distribution

Example – Probability of Corn Resolving

4.3 The Poisson Distribution

Example from the Literature – IV Treated Exacerbations in Patients with Cystic Fibrosis

4.4 Probability for Continuous Outcomes

4.5 The Normal Distribution

How Do We Use the Normal Distribution?

Illustrative Example – Normal Distribution – Birthweights

4.6 Reference Ranges

Worked Example – Reference Range – Birthweight

4.7 Other Distributions

t‐distribution

Chi‐squared Distribution

F‐distribution

Uniform Distribution

4.8 Points When Reading the Literature

4.9 Technical Section. Binomial Distribution

Poisson Distribution

Normal Distribution

4.10 Exercises

5 Populations, Samples, Standard Errors and Confidence Intervals

Summary

5.1 Populations

5.2 Samples

5.3 The Standard Error. Standard Error of the Mean

Properties of the Distribution of Sample Means

Properties of Standard Errors

Worked Example – Standard Error of a Mean – Birthweight of Preterm Infants

5.4 The Central Limit Theorem

5.5 Standard Errors for Proportions and Rates

Worked Example – Standard Error of a Proportion – Mortality from Gastrostomy in Patients with Amyotrophic Lateral Sclerosis (ProGas): A Prospective Cohort Study (ProGas Study Group 2015)

Worked Example – Standard Error of a Rate – Exacerbations in Patients with Cystic Fibrosis

Standard Deviation or Standard Error?

5.6 Standard Error of Differences

Worked Example – Standard Error of Difference in Means – Corn Size

Worked Example – Difference in Proportions –Haemorrhoid Recurrence at One Year (HubBLe Trial)

5.7 Confidence Intervals for an Estimate. Confidence Interval for a Mean

Worked Example – Confidence Interval for a Mean – Birthweights of Pre‐term Infants

Confidence Interval for a Proportion

Example from the Literature – Confidence Interval for a Proportion –Gastrostomy in Patients with Amyotrophic Lateral Sclerosis (ProGas)

Confidence Interval for a Rate

Worked Example – Confidence Interval for a Rate – Exacerbations in Patients with Cystic Fibrosis

5.8 Confidence Intervals for Differences

Example from the Literature – Confidence Interval for the Difference Between Two Means – Corn Size at Three Months

Worked Example – Confidence Interval for Difference Between Two Proportions – Haemorrhoid Recurrence at One Year (HubBLe Trial)

5.9 Points When Reading the Literature

5.10 Technical Details. Standard Errors

More Accurate Confidence Intervals for a Proportion

Worked Example – Confidence Interval for a Proportion – Gastrostomy in Patients with Amyotrophic Lateral Sclerosis (ProGas)

5.11 Exercises

6 Hypothesis Testing, P‐values and Statistical Inference

Summary

6.1 Introduction

6.2 The Null Hypothesis

Example: Distance Walked On an Endurance Shuttle Walking Test (ESWT) Before and After a Rehabilitation Programme in Patients with COPD (Waterhouse et al. 2010)

6.3 The Main Steps in Hypothesis Testing

Step 1: State Your Null Hypothesis (H0) and Alternative Hypothesis (HA)

Step 2: Choose a Significance Level, α, for Your Test

Step 3: Obtain the Probability of Observing Your Results, or Results More Extreme, if the Null Hypothesis is True (P‐value)

Step 4: Use Your P‐value to Make a Decision About Whether to Reject, or Not Reject, Your Null Hypothesis

Example: Distance Walked on a 6MWT Before and After a Rehabilitation Programme

Step 1: State Your Null Hypothesis (H0) and Alternative Hypothesis (HA)

Step 2 Choose a Significance Level, α, for Your Test

Step 3 Obtain the Probability of Observing Your Results, or Results More Extreme, If the Null Hypothesis is True (P‐value)

6.4 Using Your P‐value to Make a Decision About Whether to Reject, or Not Reject, Your Null Hypothesis

Example – Interpreting a P‐value – Distance Walked Before and After Exercise

What Does P< 0.001 Mean?

Is This Result Statistically Significant?

You Decide?

Summary of the Main Steps in Hypothesis Testing

6.5 Statistical Power. Type I Error, Test Size and Significance Level

Type II Error and Power

6.6 One‐sided and Two‐sided Tests

6.7 Confidence Intervals (CIs)

Example – Clinical Importance – Change in Distance Walked Before and After Exercise

Relationship Between Confidence Intervals and Statistical Significance

Clinically Important Difference

6.8 Large Sample Tests for Two Independent Means or Proportions

Large Sample Z‐Test for Comparison of Two Independent Means

Z‐test for Comparison of Two Independent Proportions

Example from the Literature – Haemorrhoidal Artery Ligation Versus Rubber Band Ligation for the Management of Symptomatic Second‐degree and Third‐degree Haemorrhoids (HubBLe Trial)

6.9 Issues with P‐values

6.10 Points When Reading the Literature

6.11 Exercises

7. Comparing Two or More Groups with Continuous Data

Summary

7.1 Introduction

7.2 Comparison of Two Groups of Paired Observations – Continuous Outcomes

Box 7.1 Paired t‐Test

Assumptions

Steps

Wilcoxon (Matched Pairs) Signed Rank Test

Box 7.2 Wilcoxon (Matched Pairs) Signed Rank Test

Assumptions

Steps

7.3 Comparison of Two Independent Groups – Continuous Outcomes

Box 7.3 Independent Two‐Sample t‐Test for Comparing Means

Assumptions

Steps

Mann–Whitney U Test

Box 7.4 Mann–Whitney U Test

Assumptions

Steps

Discrete Count Data

7.4 Comparing More than Two Groups

One‐way Analysis of Variance

The Kruskal–Wallis Test

7.5 Non‐Normal Distributions. Non‐parametric Tests

Why Not Always Use Non‐parametric Tests?

7.6 Degrees of Freedom

7.7 Points When Reading the Literature

7.8 Technical Details. Student's t‐Distribution

Welch's t‐Test

Wilcoxon Signed Rank Sum Test

Mann–Whitney U Test

Comparing More than Two Groups. One‐way Analysis of Variance

Box 7.5 One‐way Analysis of Variance (ANOVA)

Assumptions

Steps

The Kruskal–Wallis Test

Box 7.6 Kruskal–Wallis Test

Assumptions

Steps

7.9 Exercises

8 Comparing Groups of Binary and Categorical Data

Summary

8.1 Introduction

8.2 Comparison of Two Independent Groups – Binary Outcomes

Box 8.1 Chi‐squared Test for Association in r × c Contingency Tables

Assumptions

Steps

Fisher's Exact Test

Chi‐squared Test for Trend in a 2 × c Table

8.3 Comparing Risks

Comparing Groups Via the Odds Ratio

8.4 Comparison of Two Groups of Paired Observations – Categorical Outcomes

8.5 Degrees of Freedom

8.6 Points When Reading the Literature

8.7 Technical Details. Fisher's Exact Test

Worked Example: Calculation of Fisher's Exact Test

Chi‐squared Test for Trend (2 × c Table)

Chi‐squared Test for Trend in 2 × c Contingency Tables

Assumptions

Steps

Exact Test for Small Samples with Paired Binary Outcomes

Example

Confidence Interval for a Relative Risk

Worked Example

CI for an Odds Ratio

Worked Example

8.8 Exercises

9 Correlation and Linear Regression

Summary

9.1 Introduction

Example – Systolic and Diastolic Blood Pressure

9.2 Correlation

Box 9.1 Pearson's Correlation Coefficient

Assumptions

Steps

Hypothesis test for ρ

Confidence interval for ρ

When Not to Use the Correlation Coefficient

Example – Correlation Between Systolic and Diastolic Blood Pressure

Assumptions Underlying the Test of Significance

Box 9.2 Spearman's Rank Correlation Coefficient

Assumptions

Steps

Example – Spearman and Pearson Correlation Coefficients – Systolic and Diastolic Blood Pressure

9.3 Linear Regression. The Regression Line

Box 9.3 Linear Regression

Assumptions

Steps

Example – Linear Regression – Birthweight and Gestation

Tests of Significance and Confidence Intervals

Example – Linear Regression – Birthweight and Gestational Age – Hypothesis Test and Confidence Interval

Do the Assumptions Matter?

Regression and Prediction

Example – Prediction – Birthweight

9.4 Comparison of Assumptions Between Correlation and Regression

9.5 Multiple Regression. The Multiple Regression Equation

Examples of Uses of Multiple Regression

9.6 Correlation is not Causation

Association Between Two Variables: Correlation or Regression?

9.7 Points When Reading the Literature

9.8 Technical Details

Correlation Coefficient. Worked Example – Correlation Coefficient

Spearman's Rank Correlation

Linear Regression

Worked Example – Linear Regression

Worked Example – Calculation of Residuals

Technical Details: Checking the Assumptions for a Linear Regression Analysis

9.9 Exercises

10 Logistic Regression

Summary

10.1 Introduction

10.2 Binary Outcome Variable

Example – Logistic Regression – Foot Corn RCT

10.3 The Multiple Logistic Regression Equation

Example – Multiple Logistic Regression – Corn Healed and Baseline Corn Size

Calculating the Probability of an Event

Example – Estimating the Probability that an Individual Has the Outcome of Interest – Corning Healing

Example of Multiple Logistic Regression – Foot Corn RCT

Use of Logistic Regression in Case–Control Studies

Example from the Literature – Caffeinated Substances and Risk of Crashes in Long Distance Drivers: Case–Control Study

10.4 Conditional Logistic Regression

Example of Conditional Logistic Regression

Consequences of the Logistic Model

Example – Foot Corn RCT Group by Sex Interaction

Model Checking

10.5 Reporting the Results of a Logistic Regression

10.6 Additional Points When Reading the Literature When Logistic Regression Has Been Used

10.7 Technical Details. The Logistic Regression Model

10.8 The Wald Test

10.9 Evaluating the Model and its Fit: The Hosmer–Lemeshow Test

10.10 Assessing Predictive Efficiency (1): 2 × 2 Classification Table

10.11 Assessing Predictive Efficiency (2): The ROC Curve

10.12 Investigating Linearity

10.13 Exercises

11 Survival Analysis

Summary

11.1 Time to Event Data

Example from the Literature – Trial Endpoints – Children with Neuroblastoma

Censored Observations Can Arise in Three Ways

11.2 Kaplan–Meier Survival Curve

Example from the Literature – Kaplan–Meier Curves – Aim High Study

Procedure for Calculating a Kaplan–Meier Survival Curve

Worked Example – K‐M Survival Curve

11.3 The Logrank Test

Procedure for Calculating the Logrank Test Comparing Two Groups A and B

Worked Example – Logrank Test

11.4 The Hazard Ratio

Worked Example – Median ‘Survival’ Time – AIM High

Procedure for Calculating the HR when Comparing Two Groups

Worked Example – Hazard Ratio – 20 Randomly Sampled Patients from the AIM High Trial

Confidence Interval for HR

95% Confidence Interval (CI) for an HR

Worked Example – Confidence Interval for the HR – AIM High Trial

11.5 Modelling Time to Event Data

Cox Proportional Hazards Regression Model for Two Groups and No Covariates

Technical Example

Worked Example – Cox Model

Example from the Literature – Cox Multivariable Regression – AIM High Trial

11.6 Points When Reading Literature. Interpreting the Results of a Survival Analysis

Technical Details

11.7 Exercises

12 Reliability and Method Comparison Studies

Summary

12.1 Introduction

12.2 Repeatability

Coefficient of Variation

Coefficient of Variation

Example – CV – Total Steps Per Day

Intra‐class Correlation Coefficient (ICC)

Intra‐Class Correlation Coefficient

Example from the Literature – ICC – Paediatric Asthma Quality of Life Questionnaire

Comparison Between the Intraclass Correlation and Pearson's Correlation

12.3 Agreement

Observer(s) Agreement

Observer(s) Disagreement

Cohen's Kappa (κ)

Cohen's κ

Example from the Literature – Cohen's κ – Arteriovenous Malformations (AVM)

Worked Example – Cohen's κ – Pathology Review

12.4 Validity. Cronbach's Alpha (αCronbach)

Cronbach's Alpha, αCronbach

Example from the Literature – αCronbach – Patients with Pressure Ulcers (PU)

12.5 Method Comparison Studies

Example – Method Comparisons – Two Spirometers

Why Calculating the Correlation Coefficient is Inappropriate

Example – Bland and Altman Plots – Comparing Respiradyne and Vitalograph Spirometers

Method Comparisons

Example – Limits of Agreement – Two Spirometers

Example from the Literature – Limits of Agreement – Positron Emission Tomography (PET) and Endoscopic Ultrasound (EUS)

12.6 Points When Reading the Literature

12.7 Technical Details

Agreement by Chance (PChance)

Calculation of Cronbach's Alpha

12.8 Exercises

13. Evaluation of Diagnostic Tests

Summary

13.1 Introduction

13.2 Diagnostic Tests

13.3 Prevalence, Overall Accuracy, Sensitivity, and Specificity

Example of Calculations for Sensitivity and Specificity – Diagnosis of Prostate Cancer (Ahmed et al. 2017 )

13.4 Positive and Negative Predictive Values

Example of Calculations for PPV and NPV – Diagnosis of Prostate Cancer (Ahmed et al. 2017 )

13.5 The Effect of Prevalence

13.6 Confidence Intervals

13.7 Functions of a Screening and Diagnostic Test

13.8 Likelihood Ratio, Pre‐test Odds and Post‐test Odds

Example – Likelihood Ratio – MRI Scan for Diagnosing Prostate Cancer

13.9 Receiver Operating Characteristic (ROC) Curve

Example of Calculations – Diagnosis of PND Using the EPDS

Example from the Literature – ROC – Diagnosis of Postnatal Depression Using the EPDS

Analysis of ROC Curves

Discussion

13.10 Points When Reading the Literature About a Diagnostic Test

Technical Details. Bayes' Theorem

Example: Conditional Probabilities and Diagnostic Testing

13.11 Exercises

14 Observational Studies

Summary

14.1 Introduction

14.2 Risk and Rates

Risk is Defined as:

To Calculate a Rate the Following Are Needed

Incidence is Defined as:

Crude Mortality Rate

Example of Crude Mortality Rates

Age‐Specific Rates (ASRs)

Age‐Specific Rate

Direct and Indirect Age‐Standardised Rates

Standardised Mortality Ratio (SMR)

Issues in the Use of Standardisation

Incidence and Prevalence

14.3 Taking a Random Sample

14.4 Questionnaire and Form Design. Purpose of Questionnaires and Forms

Types of Questions

14.5 Cross‐sectional Surveys

Example from the Literature – Prevalence of Asthma in Greece

14.6 Non‐randomised Studies. Pre‐test/Post‐test Studies

Example from the Literature – Before‐and‐After Studies

Example from the Literature – Observational Study – Acute Myocardial Infarction

Quasi‐experimental Designs

Example from the Literature – Quasi‐experimental Study – Cancer in Gulf War Veterans

Interrupted Time Series

Example of an Interrupted Time Series

14.7 Cohort Studies

Design

Relative Risk

Example from the Literature – Cohort Study – Mortality in Slate Workers

The Population Attributable Risk

Attributable Risk

Worked Example – Attributable Risk

Why Quote a Relative Risk?

Size of Study

Problems in Interpretation of Cohort Studies

Post‐marketing Surveillance

14.8 Case–Control Studies. Design

Unmatched Study

Example from the Literature – Case–Control Study – Mobile Phone Use and Glioma

Odds Ratio

Worked Example – Odds Ratio – Mobile Phone Use and Glioma

Matched Studies

Odds Ratio for a Matched Case–Control Study

Example from the Literature – Matched Case Control Study – Adverse Childhood or Adult Experiences and Risk of Bilateral Oophorectomy

Analysis by Matching?

Selection of Controls

Confounding

Example from the Literature – Simpson's Paradox

Limitations of Case–Control Studies

14.9 Association and Causality

14.10 Modern Causality Methods and Big Data

14.11 Points When Reading the Literature

14.12 Technical Details. Confidence Interval for a Relative Risk

Worked Example

CI for an Odds Ratio

Worked Example

CI for Matched Case Control Study

Worked Example

14.13 Exercises

15 The Randomised Controlled Trial

Summary

15.1 Introduction

15.2 The Protocol

15.3 Why Randomise? Randomisation

Random Assignment and the Protocol

Historical Controls

15.4 Methods of Randomisation. Simple Randomisation

Blocked Randomisation

Stratified Randomisation

Carrying out Randomisation

15.5 Design Features. The Need for a Control Group

Treatment Choice and Follow‐up

Blind Assessment

Pilot Trials and Feasibility Studies

Design Features of Feasibility Studies and Pilot Trials

Example – External Pilot Trial – Physiotherapy Management of Lumbar Radicular Syndrome (LRS) – The POLAR Trial

Example – External Pilot Trial – Early Pulmonary Rehabilitation (EPR) in Patients with Chronic Obstructive Pulmonary Disease (COPD)

Example – Internal Pilot Trial – Endometrial Trauma in Women Undergoing in vitro Fertilisation (IVF)

Protocol Violations

‘Pragmatic’ and ‘Explanatory’ Trials

Superiority and Equivalence Trials

Design Features of Equivalence Trials

Example – Non‐inferiority – Adjuvant Treatment of Postmenopausal Women with Early Breast Cancer

Early and Late Phase Trials

15.6 Design Options. Parallel Designs

Example from the Literature – Parallel Group Trial – Reduced Nicotine Cigarettes

Cross‐over Designs

Example from the Literature – Cross‐over Trial – Nutrition in Diabetes

Cluster Randomised Controlled Trials

Example from the Literature – Cluster Design – Diabetes

Stepped Wedge Cluster Randomised Trials

Example for the Literature – Stepped Wedge Cluster Randomised Trial – SHAREHD

Factorial Trials

Example from the Literature – 2 × 2 Factorial Design – AspECT Trial

15.7 Meta‐analysis

15.8 Checklists for Design, Analysis and Reporting

Design

Checklist of Design Features

Analysis and Presentation

Checklist for Analysis and Presentation

15.9 CONSORT

15.10 Points When Reading the Literature About a Trial

15.11 Exercises

16 Sample Size Issues

Summary

16.1 Introduction. Why Sample Size Calculations?

Why Not Sample Size Calculations?

Summing Up

16.2 Study Size

Fundamental Ingredients for a Sample Size Calculation

Type I and Type II Error Rates

Standard Deviation of the Outcome Measure

Tips on Finding the Anticipated Standard Deviation of an Estimate

Control Group Response

The (Anticipated) Effect Size

Relationship Between Type I Error, Type II Error and Effect Size

Directions of Sample Size Estimates

16.3 Continuous Data

Worked Example – Simple Formula for a Continuous Variable – Behavioural Therapy

Example from the Literature of a Sample Size for a Continuous Outcome – The IPSU Trial

16.4 Binary Data

Example from the Literature of Sample Size Calculation for Binary Outcomes – Corn Plaster Trial

16.5 Prevalence

Worked Example – Sample Size – Prevalence

16.6 Subject Withdrawals

Example from the Literature – Withdrawals – The IPSU Trial

16.7 Other Aspects of Sample Size Calculations. Pilot Studies

Example from Literature of Sample Size for a Pilot or Feasibility Study – The POLAR Trial

Post‐hoc Sample Size Calculations

Other Designs

Summary

16.8 Points When Reading the Literature

16.9 Technical Details

Continuous Outcomes – Comparison of Two Means

Worked Example – Comparison of Two Means

Binary Outcomes – Comparison of Two Proportions

Worked Example – Comparison of Two Proportions

16.10 Exercises

17 Other Statistical Methods

Summary

17.1 Analysing Serial or Longitudinal Data

Three Broad Approaches to Analysing Repeated Measurements

Summarising Repeated Measurements

Time by Time Analysis

Response Feature Analysis – The Use of Summary Measures

The Area Under the Curve (AUC)

Calculating the AUC

Example from the Literature – AUC and the POLAR Trial

Other Summary Measures

Longitudinal Models

17.2 Poisson Regression

Use of an Offset

Calculating the Probability of an Event

Example from the Literature – Cystic Fibrosis the ACTif Trial

Extra‐Poisson Variation

17.3 Missing Data

Types and Patterns of Missing Data

Describing the Extent and Patterns of Missing Data

Incomplete Data

What if we Think the Data Is MNAR?

Imputation of Missing Data

Example – Imputation of Missing Data – IPSU Trial

17.4 Bootstrap Methods

A Simple Example of the Bootstrap

Bootstrap Methods for Confidence Interval Estimation

Example from the Literature

17.5 Points When Reading the Literature

17.6 Exercises

18 Meta‐analysis

Summary

18.1 Introduction

18.2 What is a Meta‐analysis?

Box 18.1 Meta‐analysis: What Does it Achieve?

18.3 Meta‐analysis Methods. Is a Meta‐analysis Appropriate? The PICOTS Approach

Fixed Effects and Random Effects Meta‐analysis

Combining the Results of Different Studies

Heterogeneity

18.4 Example: Mobile Phone Based Intervention for Smoking Cessation

18.5 Discussion. The Main Problems with Meta‐analysis

Presentation of Results

Further Reading

18.6 Technical Details. Fixed Effects

Random Effects

18.7 Exercises

19 Common Mistakes and Pitfalls

Summary

19.1 Introduction

19.2 Misleading Graphs and Tables. Two‐ or Three‐Dimensional Charts

Dynamite Plunger Plots

Supress the Origin or Change the Baseline

Presenting Data in Tables

19.3 Plotting Change Against Initial Value. Adjusting for Baseline

Worked Example – Weight Change

Example – Weight Change

Regression to the Mean

Example from the Literature – Change in Fasting Serum Cholesterol

19.4 Within Group Versus Between Group Analyses

14.4 P‐value from Paired t‐test

P‐value from Two Independent Samples t‐test

19.5 Analysing Paired Data Ignoring the Matching

19.6 Unit of Analysis

19.7 Testing for Baseline Imbalances in an RCT

19.8 Repeated Measures

Example – Postprandial Plasma Glucose Concentrations

Invalid Approaches

Example – Mean Response Over Time

Valid Approaches

Example – Liraglutide Versus Sitagliptin for Type 2 Diabetes

19.9 Clinical and Statistical Significance

19.10 Post Hoc Power Calculations

19.11 Predicting or Extrapolating Beyond the Observed Range of Data

19.12 Exploratory Data Analysis ‘Fishing Expeditions’

Multiple Comparisons

19.13 Misuse of P‐values

19.14 Points When Reading the Literature

Appendix Statistical Tables

Solutions to Multiple‐Choice Exercises. Chapter 2

Chapter 3

Chapter 4

Chapter 5

Chapter 6

Chapter 7

Chapter 8

Chapter 9

Chapter 10

Chapter 11

Chapter 12

Chapter 13

Chapter 14

Chapter 15

Chapter 16

Chapter 17

Chapter 18

References (Chapter numbers in square brackets)

Index. a

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e

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Отрывок из книги

Fifth Edition

Stephen J. Walters

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Table 2.5 Calculating the median, quartiles, and interquartile range for the corn size data.

The variance is expressed in square units and so is not a suitable measure for describing variability because it is not in the same units as the raw data. The solution is to take the square root of the variance to return to the original units. This gives us the standard deviation (usually abbreviated to SD or s) defined as:

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