Organizational Structure of This Book |
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xxiv | |
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Trial Strategy and Design |
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Enrollment, Allocation of Treatment, and Ethics |
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Measurement Outcome and Analysis and Interpretation of Results |
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Study Strategy and Design |
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Assessment of Risk Factors and Exposures |
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Measurement Outcome and Analysis and Interpretation of Results |
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Practical Guide to Data Sets |
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Foreword |
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xxvii | |
Preface |
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xxxi | |
Editors and Contributors |
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xxxiii | |
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INTERVENTIONAL STUDIES: Trial Strategy and Design |
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Noninferiority Trials: Is a New Treatment Almost as Effective as Another? |
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1 | (8) |
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Why Are Noninferiority Trials Conducted? |
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What Are the Limitations of Noninferiority Trials? |
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Why Was a Noninferiority Trial Conducted in This Case? |
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How Should the Results Be Interpreted? |
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Caveats to Consider When Looking at a Noninferiority Trial |
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Dose-Finding Trials: Optimizing Phase 2 Data in the Drug Development Process |
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9 | (8) |
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Why Are Dose-Response Models Used? |
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What Are the Limitations of Dose-Response Modeling? |
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Why Did the Authors Use Dose-Response Modeling in This Particular Study? |
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How Should the Dose-Response Findings Be Interpreted in This Particular Study? |
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Caveats to Consider When Looking at Results Based on a Dose-Response Model |
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Pragmatic Trials: Practical Answers to "Real World" Questions |
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17 | (8) |
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Why Are Pragmatic Trials Conducted? |
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Description of the Method |
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What Are the Limitations of Pragmatic Trials? |
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Why Was a Pragmatic Trial Conducted in This Case? |
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How Should the Results Be Interpreted? |
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Cluster Randomized Trials: Evaluating Treatments Applied to Groups |
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25 | (8) |
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Why Is Cluster Randomization Used? |
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What Are Limitations of Cluster Randomization? |
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Why Did the Authors Use Cluster Randomization in This Particular Study? |
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How Should Cluster Randomization Findings Be Interpreted in This Particular Study? |
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Caveats to Consider When Looking at a Cluster Randomized Trial |
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The Stepped-Wedge Clinical Trial: Evaluation by Rolling Deployment |
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33 | (8) |
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Why Is a Stepped-Wedge Clinical Trial Design Used? |
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Description of the Stepped-Wedge Clinical Trial Design |
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Limitations of the Stepped-Wedge Design |
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How Was the Stepped-Wedge Design Used? |
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How Should a Stepped-Wedge Clinical Trial Be Interpreted? |
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Sample Size Calculation for a Hypothesis Test |
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41 | (8) |
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Why Is Power Analysis Used? |
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What Are the Limitations of Power Analysis? |
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Why Did the Authors Use Power Analysis in This Particular Study? |
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How Should This Method's Findings Be Interpreted in This Particular Study? |
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Caveats to Consider When Looking at Results Based on Power Analysis |
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Minimal Clinically Important Difference: Defining What Really Matters to Patients |
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49 | (8) |
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What Are the Limitations of MCID Derivation Methods? |
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Why Did the Authors Use MCID in This Particular Study? |
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How Should MCID Findings Be Interpreted in This Particular Study? |
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Caveats to Consider When Looking at Results Based on MClDs |
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Enrollment, Allocation of Treatment, and Ethics |
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Randomization in Clinical Trials: Permuted Blocks and Stratification |
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57 | (8) |
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Explanation of the Concept |
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What Are Permuted Blocks and Stratified Randomization? |
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Why Are Permuted Blocks and Stratified Randomization Important? |
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Limitations of Permuted Block Randomization and Stratified Randomization |
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How Were These Approaches to Randomization Used? |
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How Does the Approach to Randomization Affect the Trial's Interpretation? |
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Equipoise in Research: Integrating Ethics and Science in Human Research |
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65 | (8) |
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Why Is Equipoise Important? |
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What Are the Limitations of Equipoise? |
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How Is Equipoise Applied in This Case? |
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How Does Equipoise Influence the Interpretation of the Study? |
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Measurement Outcomes and Analysis and Interpretation of Results |
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73 | (8) |
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Why Is Time-to-Event Analysis Used? |
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What Are the Limitations of the Proportional Hazards Model? |
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How Should Time-to-Event Findings Be Interpreted in This Particular Study? |
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Caveats to Consider When Looking at Results from a Time-to-Event Analysis |
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The "Utility" in Composite Outcome Measures: Measuring What Is Important to Patients |
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81 | (8) |
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Why Are Composite End Points Used in Clinical Studies? |
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Limitations of Composite End Points |
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How Were Composite End Points Used in This Study? |
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How Does the Use of a Composite End Point Affect the Interpretation of This Study? |
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Missing Data: How to Best Account for What Is Not Known |
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89 | (8) |
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Why Are These Methods Used? |
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What Are the Limitations of These Methods? |
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Why Did the Authors Use This Method in This Particular Study? |
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How Should This Method's Findings Be Interpreted in This Particular Study? |
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Caveats to Consider When Looking at the Results in This Study Based on This Method |
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The Intention-to-Treat Principle: How to Assess the True Effect of Choosing a Medical Treatment |
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97 | (8) |
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Why Is ITT Analysis Used? |
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What Are the Limitations of ITT Analysis? |
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Why Did the Authors Use ITT Analysis in This Particular Study? |
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Caveats to Consider When Looking at Results Based on ITT Analysis |
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Analyzing Repeated Measurements Using Mixed Models |
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105 | (8) |
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Why Are Mixed Models Used for Repeated Measures Data? |
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What Are the Limitations of Mixed Models? |
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Why Did the Authors Use Mixed Models in This Particular Study? |
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Caveats to Consider When Looking at Results From Mixed Models |
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Logistic Regression: Relating Patient Characteristics to Outcomes |
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113 | (8) |
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Why Is Logistic Regression Used? |
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Description of the Method |
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What Are the Limitations of Logistic Regression? |
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Why Did the Authors Use Logistic Regression in This Study? |
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How Should the Results of Logistic Regression |
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Be Interpreted in This Particular Study? |
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Caveats to Consider When Assessing the Results of a Logistic Regression Analysis |
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Logistic Regression Diagnostics: Understanding How Well a Model Predicts Outcomes |
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121 | (8) |
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Why Are Logistic Regression Model Diagnostic Used? |
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Description of the Method |
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What Are the Limitations of Logistic Regression Diagnostic? |
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Why Did the Authors Use Logistic Regression Diagnostics in |
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How Should the Results of Logistic Regression Diagnostics Be Interpreted in This Particular Study? |
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Caveats to Consider When Assessing the Results of Logistic Regression Diagnostics |
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Number Needed to Treat: Conveying the Likelihood of a Therapeutic Effect |
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129 | (8) |
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Explanation of the Concept |
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Why Is the NNT Important? |
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Limitations and Alternatives to the NNT |
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How Was the Concept of NNT Applied in This Particular Study? |
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How Should the NNT Be Interpreted in the Study by Zhao et al? |
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Multiple Comparison Procedures |
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137 | (8) |
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Why Are Multiple Comparison Procedures Used? |
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What Are the Limitations of Multiple Comparison Procedures? |
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Why Did the Authors Use Multiple Comparison Procedures in This Particular Study? |
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How Should This Method's Findings Be Interpreted in This Particular Study? |
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Caveats to Consider When Looking at Multiple Comparison Procedures |
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Confirmatory vs Exploratory |
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Gatekeeping Strategies for Avoiding False-Positive Results in Clinical Trials With Many Comparisons |
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145 | (8) |
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Why Is Serial Gatekeeping Used? |
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Description of the Method |
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What Are the Limitations of Gatekeeping Strategies? |
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How Was Gatekeeping Used in This Case? |
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How Should the Results Be Interpreted? |
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Multiple Imputation: A Flexible Tool for Handling Missing Data |
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153 | (8) |
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Why Is Multiple Imputation Used? |
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What Are the Limitations of Multiple Imputation? |
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Why Did the Authors Use Multiple Imputation in This Particular Study? |
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How Should Multiple Imputation Findings Be Interpreted in This Particular Study? |
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Caveats to Consider When Looking at Results Based on Multiple Imputation |
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Interpretation of Clinical Trials That Stopped Early |
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161 | (8) |
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Why Is Early Stopping Used? |
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What Are the Limitations of Early Stopping? |
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Why Did the Authors Use Early Stopping in This Study? |
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How Should Early Stopping Be Interpreted in This Particular Study? |
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Caveats to Consider When Looking at a Trial That Stopped Early |
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Bayesian Analysis: Using Prior Information to Interpretthe Results of Clinical Trials |
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169 | (6) |
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What Is Prior Information? |
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Why Is Prior Information Important? |
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Limitations of Prior Information |
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How Was Prior Information Used? |
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How Should the Trial Results Be Interpreted in Light of the Prior Information? |
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175 | (8) |
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What Are the Limitations of the DCA Method? |
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Why Did the Authors Use DCA in This Particular Study? |
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How Should DCA Findings Be Interpreted in This Particular Study? |
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Caveats to Consider When Looking at Results Based on DCA |
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Methods for Evaluating Changes in Health Care Policy---The Difference-in-Differences Approach |
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183 | (8) |
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Why Was the Difference-in-Differences Method Used? |
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What Are the Limitations of the Difference-in-Differences Method? |
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Why Did the Authors Use the Difference-in-Differences Method? |
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How Should the Findings Be Interpreted? |
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Caveats to Consider When Assessing the Results of a Difference-in-Differences Analysis |
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OBSERVATIONAL STUDIES Study Strategy and Design |
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Case-Control Studies: Using "Real-world" Evidence to Assess Association |
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191 | (8) |
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Explanation of the Method |
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What Are Case-Control and Nested Case-Control Studies? |
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Why Are Case-Control Studies Used? |
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Limitations of Case-Control Studies |
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How Was the Method Applied in This Case? |
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How Does the Case-Control Design Affect the Interpretation of the Study? |
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Meta-analyses Can Be Credible and Useful: A New Standard |
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199 | (8) |
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207 | (8) |
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Why Is Mendelian Randomization Used? |
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What Are the Limitations of Mendelian Randomization? |
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How Did the Authors Use Mendelian Randomization? |
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Caveats to Consider When Evaluating Mendelian Randomization Studies |
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Using the E-Value to Assess the Potential Effect of Unmeasured Confounding in Observational Studies |
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215 | (8) |
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What Are the Limitations of the E-Value? |
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Why Did the Authors Use the E-Value in This Particular Study? |
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How Should the E-Value Findings Be Interpreted in This Particular Study? |
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Caveats to Consider When Looking at Results Based on the E-Value |
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Assessment of Risk Factors and Exposures |
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Confounding by Indication in Clinical Research |
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223 | (8) |
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Addressing Confounding in Clinical Research |
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Use of Methods to Control Confounding |
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What Are the Limitations of Methods to Control for Confounding? |
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How Should the Results Be Interpreted? |
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Caveats to Consider When Interpreting an Analysis Intended to Adjust for Confounding by Indication |
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231 | (8) |
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Why Is Mediation Analysis Used? |
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Description of Mediation Analysis |
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What Are the Limitations of Mediation Analysis? |
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Why Did the Authors Use Mediation Analysis? |
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Caveats to Consider When Assessing the Results of Mediation Analysis |
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Measurement Outcome and Analysis and Interpretation of Results |
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Odds Ratios---Current Best Practice and Use |
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239 | (8) |
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Why Report Odds Ratios From Logistic Regression? |
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What Are the Limitations of Odds Ratios? |
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How Did the Authors Use Odds Ratios? |
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How Should the Findings Be Interpreted? |
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What Caveats Should the Reader Consider? |
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Marginal Effects---Quantifying the Effect of Changes in Risk Factors in Logistic Regression Models |
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247 | (8) |
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Why Are Marginal Effects Used? |
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What Are Marginal Effects? |
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What Are the Limitations of Marginal Effects? |
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How Should the Marginal Effects Be Interpreted in Cummings et al? |
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Adjusting for Covariates: A Source of False Findings in Published Research Studies |
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255 | (6) |
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Treatment Effects in Multicenter Randomized Clinical Trials |
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261 | (8) |
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Estimating Treatment Effects in Multicenter Clinical Trials |
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Why Are Differences Between Centers Considered |
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When Estimating Treatment Effects? |
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How Are Center Effects Incorporated into Estimates of Treatment Effects? |
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Limitations of Estimates of Treatment Effects from Multicenter Clinical Trials |
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How Were the Multicenter Data Analyzed in the Study by Dodick et al? |
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How Should the Results From This Study Be Interpreted? |
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269 | (8) |
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Why Were Propensity Methods Used? |
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What Are the Limitations of Propensity Score Methods? |
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Why Did the Authors Use Propensity Methods? |
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How Should the Findings Be Interpreted? |
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What Caveats Should the Reader Consider When Assessing the Results of Propensity Analyses? |
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Using Free-Response Receiver Operating Characteristic Curves to Assess the Accuracy of Machine Diagnosis of Cancer |
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277 | (8) |
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Why Are FROC Curves Used? |
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How Are FROC Curves Constructed? |
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What Are the Limitations of FROC Curves? |
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How Should the FROC Curves Be Interpreted in This Study? |
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Caveats to Consider When Looking at FROC Curves |
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Random-Effects Meta-analysis: Summarizing Evidence With Caveats |
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285 | (8) |
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Why Is Random-Effects Meta-analysis Used? |
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Description of Random-Effects Meta-analysis |
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Why Did the Authors Use Random-Effects Meta-analysis? |
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What Are Limitations of a Random-Effects Meta-analysis? |
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Caveats to Consider When Assessing the Results of a Random-Effects Meta-analysis |
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How Should the Results of a Random-Effects Meta-analysis Be Interpreted in This Particular Study? |
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Bayesian Hierarchical Models |
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293 | (8) |
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What Are the Limitations of BHMs? |
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How Were BHMs Used in This Case? |
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How Should BHMs Be Interpreted? |
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Evaluating Discrimination of Risk Prediction Models: The C Statistic |
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301 | (8) |
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Why Are C Statistics Used? |
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What Are the Limitations of the C Statistic? |
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Why Did the Authors Use C Statistics in Their Study? |
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How Should the Findings Be Interpreted? |
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Caveats to Consider When Using C Statistics to Assess Predictive Model Performance |
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Overview of Cost-effectiveness Analysis |
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309 | (8) |
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The Use of Cost-effectiveness Analysis |
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Description of Cost-effectiveness Analysis |
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Limitation in the Use of Cost-effectiveness Analysis |
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How Was the Cost-effectiveness Analysis Performed in This Study? |
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How Should the Cost-effectiveness Analysis Be Interpreted in This Study? |
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Choosing a Time Horizon in Cost and Cost-effectiveness Analyses |
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317 | (8) |
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The Use of Time Horizon in a Cost-effectiveness Analysis |
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Limitations Regarding Selection of Time Horizons |
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How Was Time Horizon Defined and Used in the Study? |
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How Does the Time Horizon Selected by Wittenborn et al Affect the Interpretation of the Study? |
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On Deep Learning for Medical Image Analysis |
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325 | (8) |
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Opening the Deep Learning Black Box |
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What Are the Limitations of Deep Learning Methods? |
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PRACTICAL GUIDE TO DATA SETS |
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A Checklist to Elevate the Science of Surgical Database Research |
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333 | (8) |
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Tips for Analyzing Large Data Sets From the JAMA Surgery Statistical Editors |
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341 | (8) |
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Study Population Considerations |
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Methodological and Sample Size Considerations |
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Data Elements and Presentation |
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Analytic and Statistical Considerations |
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Practical Guide to Surgical Data Sets: Healthcare Cost and Utilization Project National Inpatient Sample (NIS) |
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349 | (10) |
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Introduction to the Healthcare Cost and Utilization Project |
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Strengths of Administrative Data |
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Limitations of Administrative Data and the HCUP Databases |
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Administrative Data Limitations |
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Critical Methodologic Considerations |
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Unique Capabilities of HCUP |
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Practical Guide to Surgical Data Sets: Surveillance, Epidemiology, and End Results (SEER) Database |
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359 | (8) |
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Statistical Considerations |
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Practical Guide to Surgical Data Sets: Medicare Claims Data |
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367 | (8) |
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Pros and Cons of Medicare Data |
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Potential Avenues of Research |
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Comparative Effectiveness Research |
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Where to Find More Information |
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Practical Guide to Surgical Data Sets: Military Health System Tricare Encounter Data |
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375 | (8) |
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Salient and Unique Features of the Data Set |
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What Are Common Outcomes That Can Be Studied? |
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What Are the Limitations With This Data Set? |
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Statistical Considerations |
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Where to Find More Information |
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Practical Guide to Surgical Data Sets: Veterans Affairs Surgical Quality Improvement Program (VASQIP) |
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383 | (8) |
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Advent of the Veterans Affairs Surgical Quality Improvement Program |
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Utility and Unique Features of VASQIP |
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Statistical Considerations |
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Practical Guide to Surgical Data Sets: National Surgical Quality Improvement Program (NSQIP) and Pediatric NSQIP |
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391 | (8) |
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Data Elements and Considerations |
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Practical Guide to Surgical Data Sets: Metabolic and Bariatric Surgery Accreditation and Quality Program (MBSAQIP) |
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399 | (8) |
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Data Considerations for the MBSAQIP Participant Use File |
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Deidentification of Patients, Facilities, and Clinicians |
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Statistical Considerations |
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MBSAQIP PUF Advantages and Limitations |
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Practical Guide to Surgical Data Sets: National Cancer Database (NCDB) |
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407 | (8) |
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Data Element Considerations |
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Analytic and Statistical Considerations |
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Practical Guide to Surgical Data Sets: National Trauma Data Bank (NTDB) |
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415 | (8) |
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Data Compilation and Structure |
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Practical Guide to Surgical Data Sets: Society for Vascular Surgery Vascular Quality Initiative (SVSVQI) |
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423 | (8) |
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Statistical Considerations |
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Practical Guide to Surgical Data Sets: Society of Thoracic Surgeons (STS) National Database |
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431 | (8) |
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Data Element Considerations |
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Adult Cardiac Surgery Database (ACSD) |
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Congenital Heart Surgery Database |
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General Thoracic Surgery Database |
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Outcomes and Other Key Measures |
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Statistical Considerations |
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Glossary |
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439 | (28) |
Index |
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467 | |