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Praise for the First Edition
"Students from health, medical, pharmacy, and nursing will find... Introductory Biostatistics extremely useful. Difficult biostatistical concepts are made easier by simple and careful explanations..."
- Journal of Statistical Computation and Simulation
Maintaining the same accessible and hands-on presentation, Introductory Biostatistics, Second Edition continues to provide an organized introduction to basic statistical concepts commonly applied in research across the health sciences. With plenty of real-world examples, the new edition provides a practical,…mehr
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Praise for the First Edition
"Students from health, medical, pharmacy, and nursing will find... Introductory Biostatistics extremely useful. Difficult biostatistical concepts are made easier by simple and careful explanations..."
- Journal of Statistical Computation and Simulation
Maintaining the same accessible and hands-on presentation, Introductory Biostatistics, Second Edition continues to provide an organized introduction to basic statistical concepts commonly applied in research across the health sciences. With plenty of real-world examples, the new edition provides a practical, modern approach to the statistical topics found in the biomedical and public health fields.
Beginning with an overview of descriptive statistics in the health sciences, the book delivers topical coverage of probability models, parameter estimation, and hypothesis testing. Subsequently, the book focuses on more advanced topics with coverage of regression analysis, logistic regression, methods for count data, analysis of survival data, and designs for clinical trials. Featuring a thorough update, Introductory Biostatistics, Second Edition includes:
A new chapter on the use of higher order Analysis of Variance (ANOVA) in factorial and block designs
A new chapter on testing and inference methods for repeatedly measured outcomes including continuous, binary, and count outcomes
R incorporated throughout along with SAS ® , allowing readers to replicate results from presented examples with either software
Multiple additional exercises, with partial solutions available to aid comprehension of crucial concepts
Notes on Computations sections to provide further guidance on the use of software
A related website that hosts the large data sets presented throughout the book Introductory Biostatistics, Second Edition is an excellent textbook for upper-undergraduate and graduate students in introductory biostatistics courses. The book is also an ideal reference for applied statisticians working in the fields of public health, nursing, dentistry, and medicine.
Hinweis: Dieser Artikel kann nur an eine deutsche Lieferadresse ausgeliefert werden.
"Students from health, medical, pharmacy, and nursing will find... Introductory Biostatistics extremely useful. Difficult biostatistical concepts are made easier by simple and careful explanations..."
- Journal of Statistical Computation and Simulation
Maintaining the same accessible and hands-on presentation, Introductory Biostatistics, Second Edition continues to provide an organized introduction to basic statistical concepts commonly applied in research across the health sciences. With plenty of real-world examples, the new edition provides a practical, modern approach to the statistical topics found in the biomedical and public health fields.
Beginning with an overview of descriptive statistics in the health sciences, the book delivers topical coverage of probability models, parameter estimation, and hypothesis testing. Subsequently, the book focuses on more advanced topics with coverage of regression analysis, logistic regression, methods for count data, analysis of survival data, and designs for clinical trials. Featuring a thorough update, Introductory Biostatistics, Second Edition includes:
A new chapter on the use of higher order Analysis of Variance (ANOVA) in factorial and block designs
A new chapter on testing and inference methods for repeatedly measured outcomes including continuous, binary, and count outcomes
R incorporated throughout along with SAS ® , allowing readers to replicate results from presented examples with either software
Multiple additional exercises, with partial solutions available to aid comprehension of crucial concepts
Notes on Computations sections to provide further guidance on the use of software
A related website that hosts the large data sets presented throughout the book Introductory Biostatistics, Second Edition is an excellent textbook for upper-undergraduate and graduate students in introductory biostatistics courses. The book is also an ideal reference for applied statisticians working in the fields of public health, nursing, dentistry, and medicine.
Hinweis: Dieser Artikel kann nur an eine deutsche Lieferadresse ausgeliefert werden.
Produktdetails
- Produktdetails
- Verlag: Wiley & Sons
- 2. Aufl.
- Seitenzahl: 624
- Erscheinungstermin: 2. Mai 2016
- Englisch
- Abmessung: 239mm x 155mm x 38mm
- Gewicht: 953g
- ISBN-13: 9780470905401
- ISBN-10: 0470905409
- Artikelnr.: 42294550
- Herstellerkennzeichnung
- Libri GmbH
- Europaallee 1
- 36244 Bad Hersfeld
- 06621 890
- Verlag: Wiley & Sons
- 2. Aufl.
- Seitenzahl: 624
- Erscheinungstermin: 2. Mai 2016
- Englisch
- Abmessung: 239mm x 155mm x 38mm
- Gewicht: 953g
- ISBN-13: 9780470905401
- ISBN-10: 0470905409
- Artikelnr.: 42294550
- Herstellerkennzeichnung
- Libri GmbH
- Europaallee 1
- 36244 Bad Hersfeld
- 06621 890
Chap T. Le, PhD, is Distinguished Professor of Biostatistics and Director of Biostatistics and Bioinformatics at the University of Minnesota Masonic Cancer Center. He has provided statistical consulting for a variety of biomedical research projects, and he has worked on collaborations focusing on the analyses of survival and categorical data and, currently, in the areas of cancer and tobacco research. Dr. Le is the author of Health and Numbers: A Problems-Based Introduction to Biostatistics, Third Edition; Applied Categorical Data Analysis and Translational Research, Second Edition; and Applied Survival Analysis, all published by Wiley. Lynn E. Eberly, PhD, is Associate Professor in the Division of Biostatistics at the University of Minnesota. The author of more than 100 journal articles, Dr. Eberly has been a statistical collaborator in biomedical and public health research for more than 18 years. Her current research interests include methods for and applications to correlated data in neurodegenerative conditions, endocrinology, psychiatry/psychology, and cancer research.
Preface to the Second Edition xiii Preface to the First Edition xv About the Companion Website xix 1 Descriptive Methods for Categorical Data 1 1.1 Proportions 1 1.1.1 Comparative Studies 2 1.1.2 Screening Tests 5 1.1.3 Displaying Proportions 7 1.2 Rates 10 1.2.1 Changes 11 1.2.2 Measures of Morbidity and Mortality 13 1.2.3 Standardization of Rates 15 1.3 Ratios 18 1.3.1 Relative Risk 18 1.3.2 Odds and Odds Ratio 18 1.3.3 Generalized Odds for Ordered 2 × k Tables 21 1.3.4 Mantel-Haenszel Method 25 1.3.5 Standardized Mortality Ratio 28 1.4 Notes on Computations 30 Exercises 32 2 Descriptive Methods for Continuous Data 55 2.1 Tabular and Graphical Methods 55 2.1.1 One
Way Scatter Plots 55 2.1.2 Frequency Distribution 56 2.1.3 Histogram and Frequency Polygon 60 2.1.4 Cumulative Frequency Graph and Percentiles 64 2.1.5 Stem and Leaf Diagrams 68 2.2 Numerical Methods 69 2.2.1 Mean 69 2.2.2 Other Measures of Location 72 2.2.3 Measures of Dispersion 73 2.2.4 Box Plots 76 2.3 Special Case of Binary Data 77 2.4 Coefficients of Correlation 78 2.4.1 Pearson's Correlation Coefficient 80 2.4.2 Nonparametric Correlation Coefficients 83 2.5 Notes on Computations 85 Exercises 87 3 Probability and Probability Models 103 3.1 Probability 103 3.1.1 Certainty of Uncertainty 104 3.1.2 Probability 104 3.1.3 Statistical Relationship 106 3.1.4 Using Screening Tests 109 3.1.5 Measuring Agreement 112 3.2 Normal Distribution 114 3.2.1 Shape of the Normal Curve 114 3.2.2 Areas Under the Standard Normal Curve 116 3.2.3 Normal Distribution as a Probability Model 122 3.3 Probability Models for Continuous Data 124 3.4 Probability Models for Discrete Data 125 3.4.1 Binomial Distribution 126 3.4.2 Poisson Distribution 128 3.5 Brief Notes on the Fundamentals 130 3.5.1 Mean and Variance 130 3.5.2 Pair
Matched Case-Control Study 130 3.6 Notes on Computations 132 Exercises 134 4 Estimation of Parameters 141 4.1 Basic Concepts 142 4.1.1 Statistics as Variables 143 4.1.2 Sampling Distributions 143 4.1.3 Introduction to Confidence Estimation 145 4.2 Estimation of Means 146 4.2.1 Confidence Intervals for a Mean 147 4.2.2 Uses of Small Samples 149 4.2.3 Evaluation of Interventions 151 4.3 Estimation of Proportions 153 4.4 Estimation of Odds Ratios 157 4.5 Estimation of Correlation Coefficients 160 4.6 Brief Notes on the Fundamentals 163 4.7 Notes on Computations 165 Exercises 166 5 Introduction to Statistical Tests of Significance 179 5.1 Basic Concepts 180 5.1.1 Hypothesis Tests 181 5.1.2 Statistical Evidence 182 5.1.3 Errors 182 5.2 Analogies 185 5.2.1 Trials by Jury 185 5.2.2 Medical Screening Tests 186 5.2.3 Common Expectations 186 5.3 Summaries and Conclusions 187 5.3.1 Rejection Region 187 5.3.2 p Values 189 5.3.3 Relationship to Confidence Intervals 191 5.4 Brief Notes on the Fundamentals 193 5.4.1 Type I and Type II Errors 193 5.4.2 More about Errors and p Values 194 Exercises 194 6 Comparison of Population Proportions 197 6.1 One
Sample Problem with Binary Data 197 6.2 Analysis of Pair
Matched Data 199 6.3 Comparison of Two Proportions 202 6.4 Mantel-Haenszel Method 206 6.5 Inferences for General TwöWay Tables 211 6.6 Fisher's Exact Test 217 6.7 Ordered 2 × K Contingency Tables 219 6.8 Notes on Computations 222 Exercises 222 7 Comparison of Population Means 235 7.1 One
Sample Problem with Continuous Data 235 7.2 Analysis of Pair
Matched Data 237 7.3 Comparison of Two Means 242 7.4 Nonparametric Methods 246 7.4.1 Wilcoxon Rank
Sum Test 246 7.4.2 Wilcoxon Signed
Rank Test 250 7.5 One
Way Analysis of Variance 252 7.5.1 One
Way Analysis of Variance Model 253 7.5.2 Group Comparisons 258 7.6 Brief Notes on the Fundamentals 259 7.7 Notes on Computations 260 Exercises 260 8 Analysis of Variance 273 8.1 Factorial Studies 273 8.1.1 Two Crossed Factors 273 8.1.2 Extensions to More Than Two Factors 278 8.2 Block Designs 280 8.2.1 Purpose 280 8.2.2 Fixed Block Designs 281 8.2.3 Random Block Designs 284 8.3 Diagnostics 287 Exercises 291 9 Regression Analysis 297 9.1 Simple Regression Analysis 298 9.1.1 Correlation and Regression 298 9.1.2 Simple Linear Regression Model 301 9.1.3 Scatter Diagram 302 9.1.4 Meaning of Regression Parameters 302 9.1.5 Estimation of Parameters and Prediction 303 9.1.6 Testing for Independence 307 9.1.7 Analysis of Variance Approach 309 9.1.8 Some Biomedical Applications 311 9.2 Multiple Regression Analysis 317 9.2.1 Regression Model with Several Independent Variables 318 9.2.2 Meaning of Regression Parameters 318 9.2.3 Effect Modifications 319 9.2.4 Polynomial Regression 319 9.2.5 Estimation of Parameters and Prediction 320 9.2.6 Analysis of Variance Approach 321 9.2.7 Testing Hypotheses in Multiple Linear Regression 322 9.2.8 Some Biomedical Applications 330 9.3 Graphical and Computational Aids 334 Exercises 336 10 Logistic Regression 351 10.1 Simple Regression Analysis 353 10.1.1 Simple Logistic Regression Model 353 10.1.2 Measure of Association 355 10.1.3 Effect of Measurement Scale 356 10.1.4 Tests of Association 358 10.1.5 Use of the Logistic Model for Different Designs 358 10.1.6 Overdispersion 359 10.2 Multiple Regression Analysis 362 10.2.1 Logistic Regression Model with Several Covariates 363 10.2.2 Effect Modifications 364 10.2.3 Polynomial Regression 365 10.2.4 Testing Hypotheses in Multiple Logistic Regression 365 10.2.5 Receiver Operating Characteristic Curve 372 10.2.6 ROC Curve and Logistic Regression 374 10.3 Brief Notes on the Fundamentals 375 10.4 Notes on Computing 377 Exercises 377 11 Methods for Count Data 383 11.1 Poisson Distribution 383 11.2 Testing Goodness of Fit 387 11.3 Poisson Regression Model 389 11.3.1 Simple Regression Analysis 389 11.3.2 Multiple Regression Analysis 393 11.3.3 Overdispersion 402 11.3.4 Stepwise Regression 404 Exercises 406 12 Methods for Repeatedly Measured Responses 409 12.1 Extending Regression Methods Beyond Independent Data 409 12.2 Continuous Responses 410 12.2.1 Extending Regression using the Linear Mixed Model 410 12.2.2 Testing and Inference 414 12.2.3 Comparing Models 417 12.2.4 Special Cases: Random Block Designs and Multi
level Sampling 418 12.3 Binary Responses 423 12.3.1 Extending Logistic Regression using Generalized Estimating Equations 423 12.3.2 Testing and Inference 425 12.4 Count Responses 427 12.4.1 Extending Poisson Regression using Generalized Estimating Equations 427 12.4.2 Testing and Inference 428 12.5 Computational Notes 431 Exercises 432 13 Analysis of Survival Data and Data from Matched Studies 439 13.1 Survival Data 440 13.2 Introductory Survival Analyses 443 13.2.1 Kaplan-Meier Curve 444 13.2.2 Comparison of Survival Distributions 446 13.3 Simple Regression and Correlation 450 13.3.1 Model and Approach 451 13.3.2 Measures of Association 452 13.3.3 Tests of Association 455 13.4 Multiple Regression and Correlation 456 13.4.1 Proportional Hazards Model with Several Covariates 456 13.4.2 Testing Hypotheses in Multiple Regression 457 13.4.3 Time
Dependent Covariates and Applications 461 13.5 Pair
Matched Case-Control Studies 464 13.5.1 Model 465 13.5.2 Analysis 466 13.6 Multiple Matching 468 13.6.1 Conditional Approach 469 13.6.2 Estimation of the Odds Ratio 469 13.6.3 Testing for Exposure Effect 470 13.7 Conditional Logistic Regression 472 13.7.1 Simple Regression Analysis 473 13.7.2 Multiple Regression Analysis 478 Exercises 484 14 Study Designs 493 14.1 Types of Study Designs 494 14.2 Classification of Clinical Trials 495 14.3 Designing Phase I Cancer Trials 497 14.4 Sample Size Determination for Phase II Trials and Surveys 499 14.5 Sample Sizes for Other Phase II Trials 501 14.5.1 Continuous Endpoints 501 14.5.2 Correlation Endpoints 502 14.6 About Simon's TwöStage Phase II Design 503 14.7 Phase II Designs for Selection 504 14.7.1 Continuous Endpoints 505 14.7.2 Binary Endpoints 505 14.8 Toxicity Monitoring in Phase II Trials 506 14.9 Sample Size Determination for Phase III Trials 508 14.9.1 Comparison of Two Means 509 14.9.2 Comparison of Two Proportions 511 14.9.3 Survival Time as the Endpoint 513 14.10 Sample Size Determination for Case-Control Studies 515 14.10.1 Unmatched Designs for a Binary Exposure 516 14.10.2 Matched Designs for a Binary Exposure 518 14.10.3 Unmatched Designs for a Continuous Exposure 520 Exercises 522 References 529 Appendices 535 Answers to Selected Exercises 541 Index 585
Way Scatter Plots 55 2.1.2 Frequency Distribution 56 2.1.3 Histogram and Frequency Polygon 60 2.1.4 Cumulative Frequency Graph and Percentiles 64 2.1.5 Stem and Leaf Diagrams 68 2.2 Numerical Methods 69 2.2.1 Mean 69 2.2.2 Other Measures of Location 72 2.2.3 Measures of Dispersion 73 2.2.4 Box Plots 76 2.3 Special Case of Binary Data 77 2.4 Coefficients of Correlation 78 2.4.1 Pearson's Correlation Coefficient 80 2.4.2 Nonparametric Correlation Coefficients 83 2.5 Notes on Computations 85 Exercises 87 3 Probability and Probability Models 103 3.1 Probability 103 3.1.1 Certainty of Uncertainty 104 3.1.2 Probability 104 3.1.3 Statistical Relationship 106 3.1.4 Using Screening Tests 109 3.1.5 Measuring Agreement 112 3.2 Normal Distribution 114 3.2.1 Shape of the Normal Curve 114 3.2.2 Areas Under the Standard Normal Curve 116 3.2.3 Normal Distribution as a Probability Model 122 3.3 Probability Models for Continuous Data 124 3.4 Probability Models for Discrete Data 125 3.4.1 Binomial Distribution 126 3.4.2 Poisson Distribution 128 3.5 Brief Notes on the Fundamentals 130 3.5.1 Mean and Variance 130 3.5.2 Pair
Matched Case-Control Study 130 3.6 Notes on Computations 132 Exercises 134 4 Estimation of Parameters 141 4.1 Basic Concepts 142 4.1.1 Statistics as Variables 143 4.1.2 Sampling Distributions 143 4.1.3 Introduction to Confidence Estimation 145 4.2 Estimation of Means 146 4.2.1 Confidence Intervals for a Mean 147 4.2.2 Uses of Small Samples 149 4.2.3 Evaluation of Interventions 151 4.3 Estimation of Proportions 153 4.4 Estimation of Odds Ratios 157 4.5 Estimation of Correlation Coefficients 160 4.6 Brief Notes on the Fundamentals 163 4.7 Notes on Computations 165 Exercises 166 5 Introduction to Statistical Tests of Significance 179 5.1 Basic Concepts 180 5.1.1 Hypothesis Tests 181 5.1.2 Statistical Evidence 182 5.1.3 Errors 182 5.2 Analogies 185 5.2.1 Trials by Jury 185 5.2.2 Medical Screening Tests 186 5.2.3 Common Expectations 186 5.3 Summaries and Conclusions 187 5.3.1 Rejection Region 187 5.3.2 p Values 189 5.3.3 Relationship to Confidence Intervals 191 5.4 Brief Notes on the Fundamentals 193 5.4.1 Type I and Type II Errors 193 5.4.2 More about Errors and p Values 194 Exercises 194 6 Comparison of Population Proportions 197 6.1 One
Sample Problem with Binary Data 197 6.2 Analysis of Pair
Matched Data 199 6.3 Comparison of Two Proportions 202 6.4 Mantel-Haenszel Method 206 6.5 Inferences for General TwöWay Tables 211 6.6 Fisher's Exact Test 217 6.7 Ordered 2 × K Contingency Tables 219 6.8 Notes on Computations 222 Exercises 222 7 Comparison of Population Means 235 7.1 One
Sample Problem with Continuous Data 235 7.2 Analysis of Pair
Matched Data 237 7.3 Comparison of Two Means 242 7.4 Nonparametric Methods 246 7.4.1 Wilcoxon Rank
Sum Test 246 7.4.2 Wilcoxon Signed
Rank Test 250 7.5 One
Way Analysis of Variance 252 7.5.1 One
Way Analysis of Variance Model 253 7.5.2 Group Comparisons 258 7.6 Brief Notes on the Fundamentals 259 7.7 Notes on Computations 260 Exercises 260 8 Analysis of Variance 273 8.1 Factorial Studies 273 8.1.1 Two Crossed Factors 273 8.1.2 Extensions to More Than Two Factors 278 8.2 Block Designs 280 8.2.1 Purpose 280 8.2.2 Fixed Block Designs 281 8.2.3 Random Block Designs 284 8.3 Diagnostics 287 Exercises 291 9 Regression Analysis 297 9.1 Simple Regression Analysis 298 9.1.1 Correlation and Regression 298 9.1.2 Simple Linear Regression Model 301 9.1.3 Scatter Diagram 302 9.1.4 Meaning of Regression Parameters 302 9.1.5 Estimation of Parameters and Prediction 303 9.1.6 Testing for Independence 307 9.1.7 Analysis of Variance Approach 309 9.1.8 Some Biomedical Applications 311 9.2 Multiple Regression Analysis 317 9.2.1 Regression Model with Several Independent Variables 318 9.2.2 Meaning of Regression Parameters 318 9.2.3 Effect Modifications 319 9.2.4 Polynomial Regression 319 9.2.5 Estimation of Parameters and Prediction 320 9.2.6 Analysis of Variance Approach 321 9.2.7 Testing Hypotheses in Multiple Linear Regression 322 9.2.8 Some Biomedical Applications 330 9.3 Graphical and Computational Aids 334 Exercises 336 10 Logistic Regression 351 10.1 Simple Regression Analysis 353 10.1.1 Simple Logistic Regression Model 353 10.1.2 Measure of Association 355 10.1.3 Effect of Measurement Scale 356 10.1.4 Tests of Association 358 10.1.5 Use of the Logistic Model for Different Designs 358 10.1.6 Overdispersion 359 10.2 Multiple Regression Analysis 362 10.2.1 Logistic Regression Model with Several Covariates 363 10.2.2 Effect Modifications 364 10.2.3 Polynomial Regression 365 10.2.4 Testing Hypotheses in Multiple Logistic Regression 365 10.2.5 Receiver Operating Characteristic Curve 372 10.2.6 ROC Curve and Logistic Regression 374 10.3 Brief Notes on the Fundamentals 375 10.4 Notes on Computing 377 Exercises 377 11 Methods for Count Data 383 11.1 Poisson Distribution 383 11.2 Testing Goodness of Fit 387 11.3 Poisson Regression Model 389 11.3.1 Simple Regression Analysis 389 11.3.2 Multiple Regression Analysis 393 11.3.3 Overdispersion 402 11.3.4 Stepwise Regression 404 Exercises 406 12 Methods for Repeatedly Measured Responses 409 12.1 Extending Regression Methods Beyond Independent Data 409 12.2 Continuous Responses 410 12.2.1 Extending Regression using the Linear Mixed Model 410 12.2.2 Testing and Inference 414 12.2.3 Comparing Models 417 12.2.4 Special Cases: Random Block Designs and Multi
level Sampling 418 12.3 Binary Responses 423 12.3.1 Extending Logistic Regression using Generalized Estimating Equations 423 12.3.2 Testing and Inference 425 12.4 Count Responses 427 12.4.1 Extending Poisson Regression using Generalized Estimating Equations 427 12.4.2 Testing and Inference 428 12.5 Computational Notes 431 Exercises 432 13 Analysis of Survival Data and Data from Matched Studies 439 13.1 Survival Data 440 13.2 Introductory Survival Analyses 443 13.2.1 Kaplan-Meier Curve 444 13.2.2 Comparison of Survival Distributions 446 13.3 Simple Regression and Correlation 450 13.3.1 Model and Approach 451 13.3.2 Measures of Association 452 13.3.3 Tests of Association 455 13.4 Multiple Regression and Correlation 456 13.4.1 Proportional Hazards Model with Several Covariates 456 13.4.2 Testing Hypotheses in Multiple Regression 457 13.4.3 Time
Dependent Covariates and Applications 461 13.5 Pair
Matched Case-Control Studies 464 13.5.1 Model 465 13.5.2 Analysis 466 13.6 Multiple Matching 468 13.6.1 Conditional Approach 469 13.6.2 Estimation of the Odds Ratio 469 13.6.3 Testing for Exposure Effect 470 13.7 Conditional Logistic Regression 472 13.7.1 Simple Regression Analysis 473 13.7.2 Multiple Regression Analysis 478 Exercises 484 14 Study Designs 493 14.1 Types of Study Designs 494 14.2 Classification of Clinical Trials 495 14.3 Designing Phase I Cancer Trials 497 14.4 Sample Size Determination for Phase II Trials and Surveys 499 14.5 Sample Sizes for Other Phase II Trials 501 14.5.1 Continuous Endpoints 501 14.5.2 Correlation Endpoints 502 14.6 About Simon's TwöStage Phase II Design 503 14.7 Phase II Designs for Selection 504 14.7.1 Continuous Endpoints 505 14.7.2 Binary Endpoints 505 14.8 Toxicity Monitoring in Phase II Trials 506 14.9 Sample Size Determination for Phase III Trials 508 14.9.1 Comparison of Two Means 509 14.9.2 Comparison of Two Proportions 511 14.9.3 Survival Time as the Endpoint 513 14.10 Sample Size Determination for Case-Control Studies 515 14.10.1 Unmatched Designs for a Binary Exposure 516 14.10.2 Matched Designs for a Binary Exposure 518 14.10.3 Unmatched Designs for a Continuous Exposure 520 Exercises 522 References 529 Appendices 535 Answers to Selected Exercises 541 Index 585
Preface to the Second Edition xiii Preface to the First Edition xv About the Companion Website xix 1 Descriptive Methods for Categorical Data 1 1.1 Proportions 1 1.1.1 Comparative Studies 2 1.1.2 Screening Tests 5 1.1.3 Displaying Proportions 7 1.2 Rates 10 1.2.1 Changes 11 1.2.2 Measures of Morbidity and Mortality 13 1.2.3 Standardization of Rates 15 1.3 Ratios 18 1.3.1 Relative Risk 18 1.3.2 Odds and Odds Ratio 18 1.3.3 Generalized Odds for Ordered 2 × k Tables 21 1.3.4 Mantel-Haenszel Method 25 1.3.5 Standardized Mortality Ratio 28 1.4 Notes on Computations 30 Exercises 32 2 Descriptive Methods for Continuous Data 55 2.1 Tabular and Graphical Methods 55 2.1.1 One
Way Scatter Plots 55 2.1.2 Frequency Distribution 56 2.1.3 Histogram and Frequency Polygon 60 2.1.4 Cumulative Frequency Graph and Percentiles 64 2.1.5 Stem and Leaf Diagrams 68 2.2 Numerical Methods 69 2.2.1 Mean 69 2.2.2 Other Measures of Location 72 2.2.3 Measures of Dispersion 73 2.2.4 Box Plots 76 2.3 Special Case of Binary Data 77 2.4 Coefficients of Correlation 78 2.4.1 Pearson's Correlation Coefficient 80 2.4.2 Nonparametric Correlation Coefficients 83 2.5 Notes on Computations 85 Exercises 87 3 Probability and Probability Models 103 3.1 Probability 103 3.1.1 Certainty of Uncertainty 104 3.1.2 Probability 104 3.1.3 Statistical Relationship 106 3.1.4 Using Screening Tests 109 3.1.5 Measuring Agreement 112 3.2 Normal Distribution 114 3.2.1 Shape of the Normal Curve 114 3.2.2 Areas Under the Standard Normal Curve 116 3.2.3 Normal Distribution as a Probability Model 122 3.3 Probability Models for Continuous Data 124 3.4 Probability Models for Discrete Data 125 3.4.1 Binomial Distribution 126 3.4.2 Poisson Distribution 128 3.5 Brief Notes on the Fundamentals 130 3.5.1 Mean and Variance 130 3.5.2 Pair
Matched Case-Control Study 130 3.6 Notes on Computations 132 Exercises 134 4 Estimation of Parameters 141 4.1 Basic Concepts 142 4.1.1 Statistics as Variables 143 4.1.2 Sampling Distributions 143 4.1.3 Introduction to Confidence Estimation 145 4.2 Estimation of Means 146 4.2.1 Confidence Intervals for a Mean 147 4.2.2 Uses of Small Samples 149 4.2.3 Evaluation of Interventions 151 4.3 Estimation of Proportions 153 4.4 Estimation of Odds Ratios 157 4.5 Estimation of Correlation Coefficients 160 4.6 Brief Notes on the Fundamentals 163 4.7 Notes on Computations 165 Exercises 166 5 Introduction to Statistical Tests of Significance 179 5.1 Basic Concepts 180 5.1.1 Hypothesis Tests 181 5.1.2 Statistical Evidence 182 5.1.3 Errors 182 5.2 Analogies 185 5.2.1 Trials by Jury 185 5.2.2 Medical Screening Tests 186 5.2.3 Common Expectations 186 5.3 Summaries and Conclusions 187 5.3.1 Rejection Region 187 5.3.2 p Values 189 5.3.3 Relationship to Confidence Intervals 191 5.4 Brief Notes on the Fundamentals 193 5.4.1 Type I and Type II Errors 193 5.4.2 More about Errors and p Values 194 Exercises 194 6 Comparison of Population Proportions 197 6.1 One
Sample Problem with Binary Data 197 6.2 Analysis of Pair
Matched Data 199 6.3 Comparison of Two Proportions 202 6.4 Mantel-Haenszel Method 206 6.5 Inferences for General TwöWay Tables 211 6.6 Fisher's Exact Test 217 6.7 Ordered 2 × K Contingency Tables 219 6.8 Notes on Computations 222 Exercises 222 7 Comparison of Population Means 235 7.1 One
Sample Problem with Continuous Data 235 7.2 Analysis of Pair
Matched Data 237 7.3 Comparison of Two Means 242 7.4 Nonparametric Methods 246 7.4.1 Wilcoxon Rank
Sum Test 246 7.4.2 Wilcoxon Signed
Rank Test 250 7.5 One
Way Analysis of Variance 252 7.5.1 One
Way Analysis of Variance Model 253 7.5.2 Group Comparisons 258 7.6 Brief Notes on the Fundamentals 259 7.7 Notes on Computations 260 Exercises 260 8 Analysis of Variance 273 8.1 Factorial Studies 273 8.1.1 Two Crossed Factors 273 8.1.2 Extensions to More Than Two Factors 278 8.2 Block Designs 280 8.2.1 Purpose 280 8.2.2 Fixed Block Designs 281 8.2.3 Random Block Designs 284 8.3 Diagnostics 287 Exercises 291 9 Regression Analysis 297 9.1 Simple Regression Analysis 298 9.1.1 Correlation and Regression 298 9.1.2 Simple Linear Regression Model 301 9.1.3 Scatter Diagram 302 9.1.4 Meaning of Regression Parameters 302 9.1.5 Estimation of Parameters and Prediction 303 9.1.6 Testing for Independence 307 9.1.7 Analysis of Variance Approach 309 9.1.8 Some Biomedical Applications 311 9.2 Multiple Regression Analysis 317 9.2.1 Regression Model with Several Independent Variables 318 9.2.2 Meaning of Regression Parameters 318 9.2.3 Effect Modifications 319 9.2.4 Polynomial Regression 319 9.2.5 Estimation of Parameters and Prediction 320 9.2.6 Analysis of Variance Approach 321 9.2.7 Testing Hypotheses in Multiple Linear Regression 322 9.2.8 Some Biomedical Applications 330 9.3 Graphical and Computational Aids 334 Exercises 336 10 Logistic Regression 351 10.1 Simple Regression Analysis 353 10.1.1 Simple Logistic Regression Model 353 10.1.2 Measure of Association 355 10.1.3 Effect of Measurement Scale 356 10.1.4 Tests of Association 358 10.1.5 Use of the Logistic Model for Different Designs 358 10.1.6 Overdispersion 359 10.2 Multiple Regression Analysis 362 10.2.1 Logistic Regression Model with Several Covariates 363 10.2.2 Effect Modifications 364 10.2.3 Polynomial Regression 365 10.2.4 Testing Hypotheses in Multiple Logistic Regression 365 10.2.5 Receiver Operating Characteristic Curve 372 10.2.6 ROC Curve and Logistic Regression 374 10.3 Brief Notes on the Fundamentals 375 10.4 Notes on Computing 377 Exercises 377 11 Methods for Count Data 383 11.1 Poisson Distribution 383 11.2 Testing Goodness of Fit 387 11.3 Poisson Regression Model 389 11.3.1 Simple Regression Analysis 389 11.3.2 Multiple Regression Analysis 393 11.3.3 Overdispersion 402 11.3.4 Stepwise Regression 404 Exercises 406 12 Methods for Repeatedly Measured Responses 409 12.1 Extending Regression Methods Beyond Independent Data 409 12.2 Continuous Responses 410 12.2.1 Extending Regression using the Linear Mixed Model 410 12.2.2 Testing and Inference 414 12.2.3 Comparing Models 417 12.2.4 Special Cases: Random Block Designs and Multi
level Sampling 418 12.3 Binary Responses 423 12.3.1 Extending Logistic Regression using Generalized Estimating Equations 423 12.3.2 Testing and Inference 425 12.4 Count Responses 427 12.4.1 Extending Poisson Regression using Generalized Estimating Equations 427 12.4.2 Testing and Inference 428 12.5 Computational Notes 431 Exercises 432 13 Analysis of Survival Data and Data from Matched Studies 439 13.1 Survival Data 440 13.2 Introductory Survival Analyses 443 13.2.1 Kaplan-Meier Curve 444 13.2.2 Comparison of Survival Distributions 446 13.3 Simple Regression and Correlation 450 13.3.1 Model and Approach 451 13.3.2 Measures of Association 452 13.3.3 Tests of Association 455 13.4 Multiple Regression and Correlation 456 13.4.1 Proportional Hazards Model with Several Covariates 456 13.4.2 Testing Hypotheses in Multiple Regression 457 13.4.3 Time
Dependent Covariates and Applications 461 13.5 Pair
Matched Case-Control Studies 464 13.5.1 Model 465 13.5.2 Analysis 466 13.6 Multiple Matching 468 13.6.1 Conditional Approach 469 13.6.2 Estimation of the Odds Ratio 469 13.6.3 Testing for Exposure Effect 470 13.7 Conditional Logistic Regression 472 13.7.1 Simple Regression Analysis 473 13.7.2 Multiple Regression Analysis 478 Exercises 484 14 Study Designs 493 14.1 Types of Study Designs 494 14.2 Classification of Clinical Trials 495 14.3 Designing Phase I Cancer Trials 497 14.4 Sample Size Determination for Phase II Trials and Surveys 499 14.5 Sample Sizes for Other Phase II Trials 501 14.5.1 Continuous Endpoints 501 14.5.2 Correlation Endpoints 502 14.6 About Simon's TwöStage Phase II Design 503 14.7 Phase II Designs for Selection 504 14.7.1 Continuous Endpoints 505 14.7.2 Binary Endpoints 505 14.8 Toxicity Monitoring in Phase II Trials 506 14.9 Sample Size Determination for Phase III Trials 508 14.9.1 Comparison of Two Means 509 14.9.2 Comparison of Two Proportions 511 14.9.3 Survival Time as the Endpoint 513 14.10 Sample Size Determination for Case-Control Studies 515 14.10.1 Unmatched Designs for a Binary Exposure 516 14.10.2 Matched Designs for a Binary Exposure 518 14.10.3 Unmatched Designs for a Continuous Exposure 520 Exercises 522 References 529 Appendices 535 Answers to Selected Exercises 541 Index 585
Way Scatter Plots 55 2.1.2 Frequency Distribution 56 2.1.3 Histogram and Frequency Polygon 60 2.1.4 Cumulative Frequency Graph and Percentiles 64 2.1.5 Stem and Leaf Diagrams 68 2.2 Numerical Methods 69 2.2.1 Mean 69 2.2.2 Other Measures of Location 72 2.2.3 Measures of Dispersion 73 2.2.4 Box Plots 76 2.3 Special Case of Binary Data 77 2.4 Coefficients of Correlation 78 2.4.1 Pearson's Correlation Coefficient 80 2.4.2 Nonparametric Correlation Coefficients 83 2.5 Notes on Computations 85 Exercises 87 3 Probability and Probability Models 103 3.1 Probability 103 3.1.1 Certainty of Uncertainty 104 3.1.2 Probability 104 3.1.3 Statistical Relationship 106 3.1.4 Using Screening Tests 109 3.1.5 Measuring Agreement 112 3.2 Normal Distribution 114 3.2.1 Shape of the Normal Curve 114 3.2.2 Areas Under the Standard Normal Curve 116 3.2.3 Normal Distribution as a Probability Model 122 3.3 Probability Models for Continuous Data 124 3.4 Probability Models for Discrete Data 125 3.4.1 Binomial Distribution 126 3.4.2 Poisson Distribution 128 3.5 Brief Notes on the Fundamentals 130 3.5.1 Mean and Variance 130 3.5.2 Pair
Matched Case-Control Study 130 3.6 Notes on Computations 132 Exercises 134 4 Estimation of Parameters 141 4.1 Basic Concepts 142 4.1.1 Statistics as Variables 143 4.1.2 Sampling Distributions 143 4.1.3 Introduction to Confidence Estimation 145 4.2 Estimation of Means 146 4.2.1 Confidence Intervals for a Mean 147 4.2.2 Uses of Small Samples 149 4.2.3 Evaluation of Interventions 151 4.3 Estimation of Proportions 153 4.4 Estimation of Odds Ratios 157 4.5 Estimation of Correlation Coefficients 160 4.6 Brief Notes on the Fundamentals 163 4.7 Notes on Computations 165 Exercises 166 5 Introduction to Statistical Tests of Significance 179 5.1 Basic Concepts 180 5.1.1 Hypothesis Tests 181 5.1.2 Statistical Evidence 182 5.1.3 Errors 182 5.2 Analogies 185 5.2.1 Trials by Jury 185 5.2.2 Medical Screening Tests 186 5.2.3 Common Expectations 186 5.3 Summaries and Conclusions 187 5.3.1 Rejection Region 187 5.3.2 p Values 189 5.3.3 Relationship to Confidence Intervals 191 5.4 Brief Notes on the Fundamentals 193 5.4.1 Type I and Type II Errors 193 5.4.2 More about Errors and p Values 194 Exercises 194 6 Comparison of Population Proportions 197 6.1 One
Sample Problem with Binary Data 197 6.2 Analysis of Pair
Matched Data 199 6.3 Comparison of Two Proportions 202 6.4 Mantel-Haenszel Method 206 6.5 Inferences for General TwöWay Tables 211 6.6 Fisher's Exact Test 217 6.7 Ordered 2 × K Contingency Tables 219 6.8 Notes on Computations 222 Exercises 222 7 Comparison of Population Means 235 7.1 One
Sample Problem with Continuous Data 235 7.2 Analysis of Pair
Matched Data 237 7.3 Comparison of Two Means 242 7.4 Nonparametric Methods 246 7.4.1 Wilcoxon Rank
Sum Test 246 7.4.2 Wilcoxon Signed
Rank Test 250 7.5 One
Way Analysis of Variance 252 7.5.1 One
Way Analysis of Variance Model 253 7.5.2 Group Comparisons 258 7.6 Brief Notes on the Fundamentals 259 7.7 Notes on Computations 260 Exercises 260 8 Analysis of Variance 273 8.1 Factorial Studies 273 8.1.1 Two Crossed Factors 273 8.1.2 Extensions to More Than Two Factors 278 8.2 Block Designs 280 8.2.1 Purpose 280 8.2.2 Fixed Block Designs 281 8.2.3 Random Block Designs 284 8.3 Diagnostics 287 Exercises 291 9 Regression Analysis 297 9.1 Simple Regression Analysis 298 9.1.1 Correlation and Regression 298 9.1.2 Simple Linear Regression Model 301 9.1.3 Scatter Diagram 302 9.1.4 Meaning of Regression Parameters 302 9.1.5 Estimation of Parameters and Prediction 303 9.1.6 Testing for Independence 307 9.1.7 Analysis of Variance Approach 309 9.1.8 Some Biomedical Applications 311 9.2 Multiple Regression Analysis 317 9.2.1 Regression Model with Several Independent Variables 318 9.2.2 Meaning of Regression Parameters 318 9.2.3 Effect Modifications 319 9.2.4 Polynomial Regression 319 9.2.5 Estimation of Parameters and Prediction 320 9.2.6 Analysis of Variance Approach 321 9.2.7 Testing Hypotheses in Multiple Linear Regression 322 9.2.8 Some Biomedical Applications 330 9.3 Graphical and Computational Aids 334 Exercises 336 10 Logistic Regression 351 10.1 Simple Regression Analysis 353 10.1.1 Simple Logistic Regression Model 353 10.1.2 Measure of Association 355 10.1.3 Effect of Measurement Scale 356 10.1.4 Tests of Association 358 10.1.5 Use of the Logistic Model for Different Designs 358 10.1.6 Overdispersion 359 10.2 Multiple Regression Analysis 362 10.2.1 Logistic Regression Model with Several Covariates 363 10.2.2 Effect Modifications 364 10.2.3 Polynomial Regression 365 10.2.4 Testing Hypotheses in Multiple Logistic Regression 365 10.2.5 Receiver Operating Characteristic Curve 372 10.2.6 ROC Curve and Logistic Regression 374 10.3 Brief Notes on the Fundamentals 375 10.4 Notes on Computing 377 Exercises 377 11 Methods for Count Data 383 11.1 Poisson Distribution 383 11.2 Testing Goodness of Fit 387 11.3 Poisson Regression Model 389 11.3.1 Simple Regression Analysis 389 11.3.2 Multiple Regression Analysis 393 11.3.3 Overdispersion 402 11.3.4 Stepwise Regression 404 Exercises 406 12 Methods for Repeatedly Measured Responses 409 12.1 Extending Regression Methods Beyond Independent Data 409 12.2 Continuous Responses 410 12.2.1 Extending Regression using the Linear Mixed Model 410 12.2.2 Testing and Inference 414 12.2.3 Comparing Models 417 12.2.4 Special Cases: Random Block Designs and Multi
level Sampling 418 12.3 Binary Responses 423 12.3.1 Extending Logistic Regression using Generalized Estimating Equations 423 12.3.2 Testing and Inference 425 12.4 Count Responses 427 12.4.1 Extending Poisson Regression using Generalized Estimating Equations 427 12.4.2 Testing and Inference 428 12.5 Computational Notes 431 Exercises 432 13 Analysis of Survival Data and Data from Matched Studies 439 13.1 Survival Data 440 13.2 Introductory Survival Analyses 443 13.2.1 Kaplan-Meier Curve 444 13.2.2 Comparison of Survival Distributions 446 13.3 Simple Regression and Correlation 450 13.3.1 Model and Approach 451 13.3.2 Measures of Association 452 13.3.3 Tests of Association 455 13.4 Multiple Regression and Correlation 456 13.4.1 Proportional Hazards Model with Several Covariates 456 13.4.2 Testing Hypotheses in Multiple Regression 457 13.4.3 Time
Dependent Covariates and Applications 461 13.5 Pair
Matched Case-Control Studies 464 13.5.1 Model 465 13.5.2 Analysis 466 13.6 Multiple Matching 468 13.6.1 Conditional Approach 469 13.6.2 Estimation of the Odds Ratio 469 13.6.3 Testing for Exposure Effect 470 13.7 Conditional Logistic Regression 472 13.7.1 Simple Regression Analysis 473 13.7.2 Multiple Regression Analysis 478 Exercises 484 14 Study Designs 493 14.1 Types of Study Designs 494 14.2 Classification of Clinical Trials 495 14.3 Designing Phase I Cancer Trials 497 14.4 Sample Size Determination for Phase II Trials and Surveys 499 14.5 Sample Sizes for Other Phase II Trials 501 14.5.1 Continuous Endpoints 501 14.5.2 Correlation Endpoints 502 14.6 About Simon's TwöStage Phase II Design 503 14.7 Phase II Designs for Selection 504 14.7.1 Continuous Endpoints 505 14.7.2 Binary Endpoints 505 14.8 Toxicity Monitoring in Phase II Trials 506 14.9 Sample Size Determination for Phase III Trials 508 14.9.1 Comparison of Two Means 509 14.9.2 Comparison of Two Proportions 511 14.9.3 Survival Time as the Endpoint 513 14.10 Sample Size Determination for Case-Control Studies 515 14.10.1 Unmatched Designs for a Binary Exposure 516 14.10.2 Matched Designs for a Binary Exposure 518 14.10.3 Unmatched Designs for a Continuous Exposure 520 Exercises 522 References 529 Appendices 535 Answers to Selected Exercises 541 Index 585