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Statistical Methods in Diagnostic Medicine (eBook)

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2014 | 2. Auflage
John Wiley & Sons (Verlag)
978-1-118-62604-7 (ISBN)

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Statistical Methods in Diagnostic Medicine - Xiao-Hua Zhou, Nancy A. Obuchowski, Donna K. McClish
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Praise for the First Edition

' . . . the book is a valuable addition to the literature in the field, serving as a much-needed guide for both clinicians and advanced students.'-Zentralblatt MATH

A new edition of the cutting-edge guide to diagnostic tests in medical research

In recent years, a considerable amount of research has focused on evolving methods for designing and analyzing diagnostic accuracy studies. Statistical Methods in Diagnostic Medicine, Second Edition continues to provide a comprehensive approach to the topic, guiding readers through the necessary practices for understanding these studies and generalizing the results to patient populations.

Following a basic introduction to measuring test accuracy and study design, the authors successfully define various measures of diagnostic accuracy, describe strategies for designing diagnostic accuracy studies, and present key statistical methods for estimating and comparing test accuracy. Topics new to the Second Edition include:

  • Methods for tests designed to detect and locate lesions

  • Recommendations for covariate-adjustment

  • Methods for estimating and comparing predictive values and sample size calculations

  • Correcting techniques for verification and imperfect standard biases

  • Sample size calculation for multiple reader studies when pilot data are available

  • Updated meta-analysis methods, now incorporating random effects

Three case studies thoroughly showcase some of the questions and statistical issues that arise in diagnostic medicine, with all associated data provided in detailed appendices. A related web site features Fortran, SAS®, and R software packages so that readers can conduct their own analyses.

Statistical Methods in Diagnostic Medicine, Second Edition is an excellent supplement for biostatistics courses at the graduate level. It also serves as a valuable reference for clinicians and researchers working in the fields of medicine, epidemiology, and biostatistics.



Xiao-Hua Zhou, PhD, is Professor of Biostatistics at the University of Washington and Director and Research Career Scientist at the Biostatistics Unit of the Veterans Affairs Puget Sound Healthcare System. He is a Fellow of the American Statistical Association and the author of more than 100 published articles on statistical methods in diagnostic medicine and causal inferences.

Nancy A. Obuchowski, PhD, is Vice Chairperson of the Department of Quantitative Health Sciences at the Cleveland Clinic Foundation. A Fellow of the American Statistical Association, she has written more than 100 journal articles on the design and analysis of studies of screening and diagnostic tests.

Donna K. McClish, PhD, is Associate Professor and Graduate Program Director in Biostatistics at Virginia Commonwealth University. She has written more than 100 journal articles on statistical methods in epidemiology, diagnostic medicine, and health services research.


Praise for the First Edition "e; . . . the book is a valuable addition to the literature in the field, serving as a much-needed guide for both clinicians and advanced students."e; Zentralblatt MATH A new edition of the cutting-edge guide to diagnostic tests in medical research In recent years, a considerable amount of research has focused on evolving methods for designing and analyzing diagnostic accuracy studies. Statistical Methods in Diagnostic Medicine, Second Edition continues to provide a comprehensive approach to the topic, guiding readers through the necessary practices for understanding these studies and generalizing the results to patient populations. Following a basic introduction to measuring test accuracy and study design, the authors successfully define various measures of diagnostic accuracy, describe strategies for designing diagnostic accuracy studies, and present key statistical methods for estimating and comparing test accuracy. Topics new to the Second Edition include: Methods for tests designed to detect and locate lesions Recommendations for covariate-adjustment Methods for estimating and comparing predictive values and sample size calculations Correcting techniques for verification and imperfect standard biases Sample size calculation for multiple reader studies when pilot data are available Updated meta-analysis methods, now incorporating random effects Three case studies thoroughly showcase some of the questions and statistical issues that arise in diagnostic medicine, with all associated data provided in detailed appendices. A related web site features Fortran, SAS , and R software packages so that readers can conduct their own analyses. Statistical Methods in Diagnostic Medicine, Second Edition is an excellent supplement for biostatistics courses at the graduate level. It also serves as a valuable reference for clinicians and researchers working in the fields of medicine, epidemiology, and biostatistics.

Xiao-Hua Zhou, PhD, is Professor of Biostatistics at the University of Washington and Director and Research Career Scientist at the Biostatistics Unit of the Veterans Affairs Puget Sound Healthcare System. He is a Fellow of the American Statistical Association and the author of more than 100 published articles on statistical methods in diagnostic medicine and causal inferences. Nancy A. Obuchowski, PhD, is Vice Chairperson of the Department of Quantitative Health Sciences at the Cleveland Clinic Foundation. A Fellow of the American Statistical Association, she has written more than 100 journal articles on the design and analysis of studies of screening and diagnostic tests. Donna K. McClish, PhD, is Associate Professor and Graduate Program Director in Biostatistics at Virginia Commonwealth University. She has written more than 100 journal articles on statistical methods in epidemiology, diagnostic medicine, and health services research.

List of Figures xix

List of Tables xxiii

0.1 Preface xxix

0.2 Acknowledgements xxxi

Part I. Basic Concepts and Methods

1. Introduction 3

2. Measures of Diagnostic Accuracy 13

3. Design of Diagnostic Accuracy Studies 57

4. Estimation and Hypothesis Testing in a Single Sample 103

5. Comparing the Accuracy of Two Diagnostic Tests 165

6. Sample Size Calculations 193

7. Introduction to Meta-analysis for Diagnostic Accuracy Studies 231

Part II. Advanced Methods

8. Regression Analysis for Independent ROC Data 263

9. Analysis of Multiple Reader and/or Multiple Test Studies 297

10. Methods for Correcting Verification Bias 329

11. Methods for Correcting Imperfect Gold Standard Bias 389

12. Statistical Analysis for Meta-analysis 435

Appendix A. Case Studies and Chapter 8 Data 449

Appendix B. Jackknife and Bootstrap Methods of Estimating Variances and Confidence Intervals 477

"The authors, overall, have done a good job of revising their first edition, addressing the critical reviews as well as expanding and updating their coverage . . . In summary, this is a good book, focusing on medical diagnosis as the name promises, presenting a wealth of methods in detail with good discussion." (Journal of Biopharmaceutical Statistics, 2011)

"Early chapters are accessible to readers with a basic knowledge of statistical and medical terminology, and the second section addresses data analysts with basic training in biostatistics. Later chapters assume deeper background in statistics, but the examples should be accessible to all. The 2002 edition has been updated throughout, and three new case studies have been added." (Booknews, 1 June 2011)

LIST OF TABLES

2.1 Basic 2x2 Count Table
2.2 2x2 Probability Table
2.3 CAD-aided CTC Results of 25 Patients With and 5 Without Colon Polyps
2.4 Gap Measurements of 10 Patients With and 10 Without Fractured Heart Valve
2.5 Estimates of Se and Sp From Heart Valve Imaging Study
2.6 SPECT/CT Using 5-Category Scale from Table A.l in Appendix
2.7 CTC Results of 5250 Patients
2.8 Estimates of Accuracy for Case Study 1
2.9 ROC Areas for Some Diagnostic Tests
2.10 Estimating LR(t) from Heart Valve Imaging Study
3.1 Steps in Designing Diagnostic Accuracy Studies
3.2 Phases in Assessment of a Test’s Diagnostic Accuracy
3.3 Possible Sampling Plans for Case Study 3 (Phase III)
3.4 Common Biases in Studies of Diagnostic Test Accuracy
3.5 Example Illustrating the Typical Effect of an Imperfect Gold Standard on the Estimates of Accuracy
3.6 Verification Status of 87 Patients with Suspected Graft Infection
3.7 Accuracy Data for 39 Surgical Patients. Estimated Se=0.80, Sp=0.71
3.8 Accuracy Data for 87 Patients
3.9 Data Set-Up for Traditional MRMC Design
3.10 Data Set-Up for Unpaired-Patient, Unpaired-Reader MRMC Design
3.11 Data Set-Up for Unpaired-Patient, Paired-Reader MRMC Design
3.12 Data Set-Up for Paired-Patient, Unpaired-Reader MRMC Design
3.13 Two Common Study Designs for Computer-Aided Detection (CAD) Studies
3.14 Steps in Determining When Covariate Adjustment is Needed
4.1 Display of Binary Data
4.2 CAD Enhanced Computed Tomography Colonography Results for Detection of Colon Polyps (Reader 1)
4.3 Display of CK Results (2 categories) for Diagnosis of AMI
4.4 Display of CK Results (5 categories) for Diagnosis of AMI
4.5 Likelihood Ratio Results for AMI
4.6 Results of Magnetic Resonance Angiography (MRA) to Detect Significant Carotid Stenosis, Radiologist 4
4.7 Display of Ordinal Data
4.8 Estimation of ROC Curve Points for Smooth ROC Curve
4.9 Data for 60 Patients with Severe Head Trauma
4.10 Descriptive Data for CK-BB Measurements in 60 Severe Head Trauma Patients with Good or Poor Outcome
4.11 Estimation of Parameters and the Total Area under the ROC Curve for the CK-BB Test to Predict Poor Outcome of Severe Head Trauma
4.12 Optimal Operating Points: Using the CK-BB Test to Predict Poor Outcomes of Severe Head Trauma as a Function of the Prevalence and Relative Costs
5.1 Display of Unpaired Binary-Scale Data to Compare Sensitivity of Two Medical Tests (Subjects with the Condition)
5.2 Display of Paired Binary Data to Compare Sensitivity, Specificity and Predictive Values of Two Medical Tests
5.3 Paired Test Results of Radiologists 3 and 4 for Using MRA to Detect Significant Carotid Stenosis
5.4 Computed Tomography Colonography (CTC) Without CAD Enhancement, for Detection of Colon Polyps with Reader 1
5.5 Parameter Estimates, Variances and Covariances for CK-BB Enzyme Data for Younger and Older Head Trauma Patients
5.6 Paired Test Results for MRA Detection of Significant Carotid Stenosis
5.7 Variance Covariance Matrix of Parameter Estimates for MRA Example
6.1 Pairs of Binormal Parameters for Different Values of the ROC Area
6.2 Number of Patients with the Condition Required for Comparing the Same Two ROC Curves But Using Different Measures of Accuracy.
6.3 Parameters Needed for Planning Multi-Reader ROC Study
6.4 Parameter Values in Various MRMC Study Designs
6.5 Estimated Power for Various MRMC Study Designs and Sample Sizes.
7.1 Data for 14 Studies of Duplex and Color Guided Duplex Ultrasonography to Detect Serious Stenosis.
7.2 Worksheet to Construct SROC Curves for Comparing Duplex and Color Guided Duplex Ultrasonography to Detect Serious Stenosis.
7.3 Results of Estimating Regression Equation for Comparing Regular and Color-Guided Duplex US to Detect Serious Stenosis
7.4 Summary of Seven Studies of the Dexamethasone Suppression Test.
8.1 Ultrasound Rating Data By the Four Radiologists
8.2 Estimated Parameters and Standard Errors in the Hearing Test Example
8.3 Parameter Estimates and the Associated Standard Deviations in the Ordinal Regression Model for Ultrasound Rating Data
9.1 Estimates for Cutoff Points, Location and Scale Parameters in Marginal Ordinal Regression Model (9.13)
9.2 95% CI’s for AUC’s for the Three MR Imaging Techniques by Institution for the Prostate Cancer Example
9.3 Parameter Estimates with Bootstrapped 95%CI
9.4 Estimated AUC’s and the Differences in AUC’s with Bootstrapped 95%CI
9.5 Analysis of Variance Table for Mixed Effect Model (9.27)
10.1 Observed Data for a Single Binary Scale Test
10.2 Hepatic Scintigraph Data
10.3 Observed Data for Two Paired Binary Scale Tests
10.4 Paired Screening Tests for Dementia Among 75 or Older Subjects in Indianapolis
10.5 Observed Data for Two Paired Binary Scale Tests at the gth Covariate Pattern (X = xg)
10.6 Data from the Alzheimer study: Entries are Numbers of Subjects
10.7 Observed Data for a Single Ordinal Scale Test
10.8 CT Data of 53 Patients with Fever of Uncertain Origin
10.9 Observed Data for a Single Ordinal Scale Test at the gth Covariate Pattern (X = xg)
10.10 New Screening Test for Dementia Disorder Data
10.11 Area under the ROC Curve of the Ordinal Scale Screening Test
10.12 P-Values for the Null Hypothesis That There Are No Differences in ROC Areas
10.13 Observed Data for Paired Ordinal Scale Tests at the gth Covariate Pattern
10.14 The Clinical Assessment (T) Data in the NACC MDS Example
10.15 The Estimated Location and Scale Parameters with the Associated Standard Errors for the NACC MDS Clinical Diagnosis Data under the MAR Verification Assumption
10.16 MRI and CT Data in Staging Pancreatic Cancer Example
10.17 The Estimated Location and Scale Parameters with the Associated Standard Errors for the MMSE Test Using the NACC MDS Data
10.18 Estimated Model Parameters and Their Standard Errors for the MMSE Data under a Non-MAR Verification Model. (Significant Coefficients are in Bold Font.)
11.1 Results of a New Test (T) and an Imperfect Gold Standard (R) with Known Sensitivity of 90% and Specificity of 70%
11.2 Conditional Joint Probability of a New Test (T) and an Imperfect Gold Standard (R) Given the Condition Status (D)394
11.3 Classification of Results by T and R Based on the Conditional Joint Probability Given in Table (11.2)
11.4 A General Data Structure For a Single Test and Imperfect Gold Standard
11.5 Results by Stool Examination (T) and Serologic Test (R) for Strongyloides Infection
11.6 Results by Stool Examination (T) and Serologic test (R) For Strongyloides Infection
11.7 Marginal Prior and Posterior Medians and Equal-tailed 95% Credible Intervals in Strongyloides Infection Example Under the CIA
11.8 Marginal Prior and Posterior Medians and Equal-tailed 95% Credible Intervals in Strongyloides Infection Example Under a Conditional Dependence Model
11.9 Results of Mantoux and Tine Tests for Tuberculosis in Two Populations
11.10 Parameter Estimates of Mantoux and Tine Tests for Tuberculosis in Two populations Using the ML Approach Under the CIA
11.11 Bayesian Results for the Accuracy of Mantoux and Tine Tests Under the CIA
11.12 Bayesian Estimates of the Accuracy of Mantoux and Tine Tests for Tuberculosis Assuming a Conditional Dependence Model
11.13 Assessments of Pleural Thickening by Three...

Erscheint lt. Verlag 21.8.2014
Reihe/Serie Wiley Series in Probability and Statistics
Wiley Series in Probability and Statistics
Wiley Series in Probability and Statistics
Sprache englisch
Themenwelt Mathematik / Informatik Mathematik Statistik
Mathematik / Informatik Mathematik Wahrscheinlichkeit / Kombinatorik
Studium 2. Studienabschnitt (Klinik) Anamnese / Körperliche Untersuchung
Studium Querschnittsbereiche Epidemiologie / Med. Biometrie
Technik
Schlagworte Biostatistics • Biostatistik • Diagnostik • Medical Science • Medical Statistics & Epidemiology • Medizin • Medizinische Statistik u. Epidemiologie • statistical methods in diagnostic medicine, diagnostic studies, diagnostic study design, diagnostic study analysis, diagnostic accuracy data, diagnostic test accuracy, diagnostic medicine research, FORTRAN program listings, statistical research in diagnostic medicine, meta-analysis methods, diagnostic accuracy studies • Statistics • Statistik
ISBN-10 1-118-62604-4 / 1118626044
ISBN-13 978-1-118-62604-7 / 9781118626047
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