Biostatistics Decoded

ISBN-13: 9781119584209 / Angielski / Twarda / 2020 / 480 str.

A. Gouveia Oliveira

Spis treści

Preface xi1 Populations and Samples 11.1 The Object of Biostatistics 11.2 Scales of Measurement 31.3 Central Tendency Measures 51.4 Sampling 81.5 Inferences from Samples 111.6 Measures of Location and Dispersion 141.7 The Standard Deviation 151.8 The n . 1 Divisor 161.9 Degrees of Freedom 181.10 Variance of Binary Variables 191.11 Properties of Means and Variances 201.12 Descriptive Statistics 221.13 Sampling Variation 251.14 The Normal Distribution 271.15 The Central Limit Theorem 291.16 Properties of the Normal Distribution 301.17 Probability Distribution of Sample Means 321.18 The Standard Error of the Mean 331.19 The Value of the Standard Error 351.20 Distribution of Sample Proportions 371.21 Convergence of Binomial to Normal Distribution 392 Descriptive Studies 412.1 Designing a Research 412.2 Study Design 422.3 Classification of Descriptive Studies 442.4 Cross-sectional Studies 452.5 Inferences from Means 472.6 Confidence Intervals 482.7 Statistical Tables 492.8 The Case of Small Samples 512.9 Student's t Distribution 542.10 Statistical Tables of the t Distribution 562.11 Inferences from Proportions 582.12 Statistical Tables of the Binomial Distribution 602.13 Sample Size Requirements 612.14 Longitudinal Studies 632.15 Incidence Studies 652.16 Cohort Studies 662.17 Inference from Incidence Studies 702.18 Standardization 722.19 Time-to-Event Cohort Studies 752.20 The Actuarial Method 762.21 The Kaplan-Meier Method 792.22 Probability Sampling 822.23 Simple Random Sampling 842.24 Replacement in Sampling 852.25 Stratified Sampling 872.26 Multistage Sampling 923 Analytical Studies 973.1 Objectives of Analytical Studies 973.2 Measures of Association 983.3 Odds, Logits, and Odds Ratios 993.4 Attributable Risk 1013.5 Classification of Analytical Studies 1033.6 Uncontrolled Analytical Studies 1043.7 Comparative Analytical Studies 1053.8 Hybrid Analytical Studies 1093.9 Non-probability Sampling in Analytical Studies 1113.10 Comparison of Two Means 1113.11 Comparison of Two Means from Small Samples 1143.12 Comparison of Two Proportions 1164 Statistical Tests 1214.1 The Null and Alternative Hypotheses 1214.2 The z-Test 1224.3 The p-Value 1254.4 Student's t-Test 1264.5 The Binomial Test 1284.6 The Chi-Square Test 1304.7 The Table of the Chi-Square Distribution 1344.8 Analysis of Variance 1354.9 Partitioning the Sum of Squares 1394.10 Statistical Tables of the F Distribution 1424.11 The ANOVA Table 1435 Aspects of Statistical Tests 1455.1 One-Sided Tests 1455.2 Power of a Statistical Test 1495.3 Sample Size Estimation 1505.4 Multiple Comparisons 1535.5 Scale Transformation 1555.6 Non-parametric Tests 1566 Cross-sectional Studies 1616.1 Linear Regression 1616.2 The Least Squares Method 1636.3 Linear Regression Estimates 1666.4 Regression and Correlation 1716.5 The F-Test in Linear Regression 1736.6 Interpretation of Regression Analysis Results 1766.7 Multiple Regression 1776.8 Regression Diagnostics 1806.9 Selection of Predictor Variables 1846.10 Independent Nominal Variables 1856.11 Interaction 1886.12 Nonlinear Regression 1907 Case-Control Studies 1937.1 Analysis of Case-Control Studies 1937.2 Logistic Regression 1947.3 The Method of Maximum Likelihood 1967.4 Estimation of the Logistic Regression Model 1987.5 The Likelihood Ratio Test 2017.6 Interpreting the Results of Logistic Regression 2027.7 Regression Coefficients and Odds Ratios 2037.8 Applications of Logistic Regression 2047.9 The ROC Curve 2057.10 Model Validation 2088 Cohort Studies 2138.1 Repeated Measurements 2138.2 The Paired t-Test 2138.3 McNemar's Test 2158.4 Generalized Linear Models 2168.5 The Logrank Test 2198.6 The Adjusted Logrank Test 2228.7 The Incidence Rate Ratio 2248.8 The Cox Proportional Hazards Model 2258.9 Assumptions of the Cox Model 2298.10 Interpretation of Cox Regression 2309 Measurement 2339.1 Construction of Clinical Questionnaires 2339.2 Factor Analysis 2349.3 Interpretation of Factor Analysis 2379.4 Factor Rotation 2399.5 Factor Scores 2419.6 Reliability 2429.7 Concordance 2489.8 Validity 2539.9 Validation of Diagnostic Tests 25510 Experimental Studies 25710.1 Main Design Features and Classification 25710.2 Experimental Controls 26010.3 Replicates 26110.4 Classification of Experimental Designs 26210.5 Completely Randomized Design 26310.6 Interaction 26810.7 Full Factorial Design 26910.8 The Random Effects Model 27410.9 Components of Variance 27510.10 ANOVA Model II and Model III 27810.11 Rules for the Definition of the Error Terms 28210.12 ANOVA on Ranks 28411 Blocking 28511.1 Randomized Block Design 28511.2 Generalized Randomized Block Design 28811.3 Incomplete Block Design 29111.4 Factorial Design with Randomized Blocks 29211.5 Latin and Greco-Latin Square Design 29312 Simultaneous Inference 29712.1 Multiple Comparisons 29712.2 Generalist Methods 29812.3 Multiple Comparisons of Group Means 30312.4 Pairwise Comparison of Means 30412.5 Different Variances 31212.6 Comparison to a Control 31312.7 Comparison of post hoc Tests 31512.8 Complex Comparisons 31612.9 Tests of Multiple Contrasts 32012.10 A posteriori Contrasts 32412.11 The Size of an Experiment 32613 Factorial ANOVA 32913.1 The n-Way ANOVA 32913.2 The 2^k Factorial Design 33113.3 The 2^k Factorial Design with Blocking 33513.4 The Fractional Factorial Design 33714 Nested Designs 33914.1 Split-Plot Design 33914.2 Nested (Hierarchical) Design 34314.3 Mixed Model Nested ANOVA 34514.4 Mixed Model Nested ANOVA with Three Sublevels 34914.5 Pure Model II Nested ANOVA 35215 Repeated Measures 35515.1 Repeated Measures ANOVA 35515.2 Repeated Measures ANOVA with Two Factors 35915.3 ANOVA with Several Repeated Measures 36115.4 Multivariate Tests 36216 Clinical Trials 36316.1 Classification of Clinical Trials 36316.2 The Clinical Trial Population 36516.3 The Efficacy Criteria 36616.4 Controlled Clinical Trials 36716.5 The Control Group 36916.6 Blinding 37016.7 Randomization 37116.8 Non-comparative Clinical Trials 37516.9 Regression Toward the Mean 37816.10 Non-randomized Controlled Clinical Trials 37916.11 Classical Randomized Clinical Trial Designs 38116.12 Alternative Clinical Trial Designs 38516.13 Pragmatic Clinical Trials 38716.14 Cluster Randomized Trials 38916.15 The Size of a Clinical Trial 39316.16 Non-inferiority Clinical Trials 39816.17 Adaptive Clinical Trials 40316.18 Group Sequential Plans 40516.19 The Alpha Spending Function 40716.20 The Clinical Trial Protocol 40916.21 The Data Record 41117 Analysis of Clinical Trials 41317.1 General Analysis Plan 41317.2 Data Preparation 41417.3 Study Populations 41517.4 Primary Efficacy Analysis 41817.5 Analysis of Multiple Endpoints 42017.6 Secondary Analyses 42317.7 Safety Analysis 42418 Meta-analysis 42718.1 Purpose of Meta-analysis 42718.2 Measures of Effect 42818.3 The Inverse Variance Method 42918.4 The Random Effects Model 43518.5 Heterogeneity 43918.6 Publication Bias 44218.7 The Forest Plot 444References 447Index 455

O autorze

A. GOUVEIA OLIVEIRA is a M.D. with a PhD in Biostatistics from the University of Lisbon, Portugal. For the last 20 years, he has been dedicated to clinical and basic research. He was the founder and CEO of Datamedica, a full-service Contract Research Organization based in Lisbon, Portugal, where he designed, conducted and analyzed a large number of epidemiologic studies and clinical trials for all the major pharmaceutical companies. He was Associate Professor of Biomedical Informatics at the Medical University of South Carolina, USA, and currently Associate Professor of Biostatistics in the Pharmaceutical Sciences Department of the Federal University of Rio Grande do Norte in Natal, Brazil. He is the author or co-author of over 100 papers published in leading scientific journals.