Biostatistics for Medical and Biomedical Practitioners

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Basic Biostatistics for Medical and Biomedical Practitioners, Second Edition makes it easier to plan experiments, with an emphasis on sample size. It also shows what choices are available when simple tests are unsuitable and offers investigators an overview of how the kinds of complex tests that they won't do on their own work. The second edition presents a new, revised and enhanced version of the chapters, taking into consideration new developments and tools available, discussing topics, such as the basic aspects of statistics, continuous distributions, hypothesis testing, discrete distributions, probability in epidemiology and medical diagnosis, comparing means, regression and correlation. This book is a valuable source for students and researchers looking to expand or refresh their understanding of statistics as it applies to the biomedical and research fields. Based on the author’s 40+ years of teaching statistics to medical fellows and biomedical researchers across a wide range of fields, it is a valuable source for researchers who need to understand more about biostatistics to apply it to their work.

Author(s): Julien Hoffman
Edition: 2
Publisher: Academic Press
Year: 2019

Language: English
Pages: 685

Cover......Page 1
BASIC BIOSTATISTICS
FOR MEDICAL AND
BIOMEDICAL
PRACTITIONERS
......Page 3
Copyright......Page 4
About the Author......Page 5
Preface......Page 6
References......Page 10
Acknowledgments......Page 11
Section I: Basic Aspects of Statistics
......Page 12
Populations and samples......Page 13
The effects of variability......Page 14
Variables and parameters......Page 15
Description......Page 16
Statistical inference......Page 17
General Approach to Study Design......Page 19
Replication and pseudoreplication......Page 21
A Brief History of Statistics......Page 23
References......Page 26
Early Use of Statistics......Page 28
Statistical Misuse......Page 29
Basic Guides to Statistics......Page 33
References......Page 34
Statistical Power......Page 39
Effect Size......Page 41
Posthoc Power Analysis......Page 46
Appendix......Page 47
Calculation of Power......Page 48
References......Page 50
Counting......Page 52
A Sorting Experiment......Page 53
Histograms and Frequency Polygons......Page 54
Transformations......Page 58
Stem and Leaf Diagrams......Page 59
Arithmetic Mean......Page 65
Median and Quantiles......Page 67
The Geometric Mean......Page 71
Harmonic Mean......Page 72
Measures of Variability......Page 73
Standard Deviation......Page 74
Interquartile Distance......Page 77
Tables......Page 79
Figures and Graphs......Page 80
Box Plots......Page 83
General Aspects......Page 84
Trimming and Winsorization......Page 85
Median Absolute Deviation......Page 87
Combining Experiments (Addition)......Page 88
Multiplying Means (Taylor, 1982)......Page 89
Three Useful Applications of the Variance......Page 90
References......Page 91
Types of Probability......Page 93
Basic Principles and Definitions......Page 94
Additional definitions......Page 95
Other definitions......Page 96
Conditional Probability......Page 99
Bayes Theorem......Page 100
Worked Problems......Page 102
References......Page 105
Section II: Continuous Distributions
......Page 106
Normal or Gaussian Curve......Page 107
The Quincunx......Page 110
Properties of the Normal Curve......Page 111
Description of the Distribution Shape......Page 114
Kurtosis......Page 116
Determining Normality......Page 117
Ungrouped Data......Page 122
References......Page 124
Central Limit Theorem......Page 126
Setting confidence limits......Page 130
Tolerance and Prediction Limits......Page 133
Reporting Results......Page 136
Graphic Representation......Page 137
References......Page 139
Exponential Distribution......Page 141
Logarithmic Distribution......Page 142
Chi-square Distribution......Page 143
Variance Ratio (F) Distribution......Page 144
References......Page 146
Outliers......Page 147
Slippage and contamination......Page 148
Dealing with outliers......Page 150
Extreme Values......Page 151
References......Page 152
Section III: Hypothesis Testing
......Page 154
Hypotheses......Page 155
Criticism of NHST......Page 161
Hypothesis Testing and Maximum Likelihood; the Bayes Factor......Page 163
Bayes Factor......Page 164
Type I Error and α......Page 165
References......Page 166
Statistical Power......Page 168
Effect Size......Page 170
Posthoc Power Analysis......Page 175
Appendix......Page 176
Calculation of Power......Page 177
References......Page 179
Section IV: Discrete Distributions
......Page 181
Permutations......Page 182
Combinations......Page 183
Worked Problems......Page 184
References......Page 185
General Formula......Page 186
Fisher's Exact Test (Fisher-Irwin Test)......Page 187
Reference......Page 188
Goodness of Fit......Page 189
Continuity Correction......Page 191
2x2 Contingency Tables......Page 192
Practical Matters......Page 197
Odds Ratio......Page 198
Cautionary Tales......Page 200
Larger Contingency Tables......Page 206
Fisher's Exact Test......Page 208
Determining Power and Sample Size......Page 211
Cochran-Mantel-Haenszel (C-M-H) Test and Confounders......Page 213
Testing for Trends in Proportions (Cochran-Armitage Test)......Page 216
Problems With R x C Tables......Page 218
Multidimensional Tables......Page 219
References......Page 222
Paired Samples: McNemars Test......Page 224
Testing Ordered Categorical Data: Kolmogorov-Smirnov (K-S) Tests......Page 227
Kolmogoro-Smirnov One-Sample Test......Page 228
Kolmogorov-Smirnov Two-Sample Test......Page 230
Two Categories......Page 232
Weighted Kappa......Page 234
References......Page 238
Introduction......Page 239
Bernoulli formula......Page 242
Fitting a binomial distribution to a set of trial results......Page 244
Continuity correction......Page 246
Comparing probabilities......Page 249
Exact confidence limits......Page 250
Multinomial Distribution......Page 251
References......Page 252
Proportions and Binomial Theorem......Page 253
Confidence Limits......Page 254
Sample Size......Page 255
Comparing Proportions......Page 256
References......Page 257
Introduction......Page 258
Relationship to the Binomial Distribution......Page 259
Goodness of Fit to a Poisson Distribution......Page 260
The Ratio of the Variance to the Mean of a Poisson Distribution......Page 263
Exact Method......Page 265
The Square Root Transformation......Page 266
Cumulative Poisson Probabilities......Page 267
Differences Between Means of Poisson Distributions......Page 268
Comparison of Counts Not Based on Same Units......Page 269
Comparing the Ratio of Two Poisson Variates......Page 271
Determining the Required Sample Size......Page 272
References......Page 274
Probability of r Successes......Page 275
Overdispersed Distribution......Page 277
References......Page 278
Section V: Probability in Epidemiology and Medical Diagnosis
......Page 280
Cohort study......Page 281
Noncohort study......Page 283
Sample size and power......Page 288
Attributable risk......Page 289
Population attributable risk......Page 290
Relative risks below 1; number needed to treat......Page 291
Differences between proportions......Page 293
Difference between proportional ratios (Proportional AR and Proportional PAR)......Page 294
References......Page 295
Further Reading......Page 296
Bayes Theorem Applied......Page 297
Sensitivity and Specificity......Page 300
Practical Issues of Screening Tests......Page 304
Spectrum Bias or Effect......Page 307
Likelihood Ratios......Page 309
Cutting Points......Page 313
ROC Curves......Page 316
Some Comments on Screening Tests......Page 321
References......Page 322
Section VI: Comparing Means
......Page 325
Paired t-Test......Page 326
Unpaired t-Test......Page 335
Unequal Variances......Page 338
Nonparametric or Distribution-Free Tests......Page 340
The Wilcoxon Signed Rank Test......Page 341
The Sign Test......Page 342
Requirements......Page 344
The Paired t-Test Implies an Additive Model......Page 346
Ranking Transforms......Page 348
The Meaning of the Mann-Whitney Test......Page 349
Problems......Page 350
References......Page 351
Cross-Over Trials......Page 352
Equivalence and Noninferiority Testing......Page 356
References......Page 359
Introduction......Page 360
Bonferroni Correction and Equivalent Tests......Page 361
Error Rates......Page 363
Extreme Multiplicity and False Discovery Rates......Page 365
Group Sequential Boundaries......Page 367
Sequential Analysis......Page 370
Adaptive Methods......Page 372
References......Page 373
Basic Test......Page 376
Requirements for Test......Page 382
Homogeneity of Variance......Page 384
Kruskal-Wallis Test......Page 386
Effect Size......Page 388
Sample Size......Page 389
Multiple Comparisons......Page 390
Studentized Range Test......Page 391
Single Step Tests......Page 393
Multiple Step Tests......Page 394
Recommendations......Page 398
Linear Combinations......Page 399
Planned Experiments......Page 400
References......Page 401
Introduction......Page 403
Two-Way ANOVA......Page 404
Additivity......Page 407
Friedman Test......Page 409
Cochrane's Q-Test......Page 410
Interaction......Page 411
Missing Data or Unequal Cell Sizes......Page 417
Nested Designs......Page 418
Model II ANOVA......Page 422
More About Repeated Measures Designs......Page 423
Bibliography......Page 425
Section VII: Regression and Correlation
......Page 426
Introduction......Page 427
Transforming Curves......Page 428
Model I Linear Regression Basics......Page 432
Additional Calculations......Page 441
Residuals......Page 442
Confidence Limits......Page 448
Comparison Methods (Basic)......Page 452
Heteroscedasticity......Page 457
The Comparison Problem (Advanced)......Page 459
Comparing Two or More Lines......Page 460
Outliers......Page 464
Leverage......Page 465
Influence......Page 466
Ratio Measurements and Scaling Factors......Page 468
References......Page 470
Inverse Prediction......Page 472
Line of Best Fit Passes Through Zero......Page 475
Errors in the X Variate......Page 476
Break Points......Page 477
Resistant Lines......Page 478
Derivation of Formulas for a Straight Line Passing Through Zero......Page 480
References......Page 481
Introduction......Page 482
P Values and Confidence Limits......Page 484
Sample Size and Power......Page 485
Spearman's Test......Page 492
Kendall's Tau Test......Page 493
Partial Correlation......Page 495
Spurious Correlation......Page 497
Reliability......Page 500
Rater Agreement......Page 501
Related Expressions......Page 503
References......Page 504
Multiple Linear Regression With Two X Variables......Page 506
Prerequisites for Multiple Regression......Page 508
Determining the ``Best´´ Regression......Page 509
Which Is the Best Regression?......Page 511
Nonlinear Regression: Polynomial Regression......Page 512
Conclusions About Regression Methods in General......Page 513
Multiple Regression With Many Independent X Variates......Page 514
Analysis of Cystic Fibrosis Example......Page 518
Detecting Multicollinearity......Page 519
All Subsets Regression......Page 521
Forward Selection......Page 524
Nonlinear Regression: Polynomial Regression......Page 529
Splines......Page 532
Correcting for Multicollinearity......Page 533
Principles of Nonlinear Regression......Page 535
Dummy or Indicator Variables......Page 536
Longitudinal Regression......Page 539
References......Page 540
Introduction......Page 542
Wald-Wolfowitz Runs Test......Page 543
Up-and-Down Runs Test......Page 544
Rank von Neumann Ratio......Page 545
Ratio Measurements......Page 546
Control Charts......Page 547
Cumulative Sum Techniques......Page 549
Serial Measurements......Page 553
References......Page 554
General Principles......Page 556
Quantal Dose-Response Curves......Page 558
References......Page 560
Introduction......Page 561
Single Explanatory Variable......Page 563
Multiple Explanatory Variables......Page 566
Appropriateness of Model......Page 568
References......Page 569
Introduction......Page 570
Suitability of Poisson Regression......Page 571
Correcting for Overdispersion......Page 573
References......Page 574
Section VIII: Miscellaneous Topics
......Page 575
Introduction......Page 576
Basic Method......Page 578
Confidence Limits......Page 582
Comparison of Different Survival Curves......Page 583
Sample Size......Page 584
Calculating the Log-Rank Test......Page 585
The Hazard Function......Page 586
Hazard Ratio......Page 587
Cox Proportional Hazards Regression......Page 589
Competing Risks Analysis......Page 592
References......Page 595
Forest Graphs......Page 597
Funnel plots......Page 599
Radial Plots......Page 601
LAbbé Plots......Page 602
Appendix......Page 603
References......Page 604
Bootstrap......Page 606
Permutations (or Randomization) Test......Page 609
Monte Carlo Methods......Page 610
References......Page 611
Sampling Problems......Page 613
Randomization......Page 616
Clinical Trials......Page 619
Placebo Effect......Page 624
Alternatives to Randomized Clinical Trials......Page 625
Propensity Analysis......Page 626
References......Page 627
Chapter 4......Page 630
Chapter 6......Page 631
Chapter 11......Page 634
Chapter 13......Page 635
Chapter 14......Page 636
Chapter 15......Page 640
Chapter 16......Page 643
Chapter 17......Page 646
Chapter 18......Page 648
Chapter 19......Page 651
Chapter 20......Page 652
Chapter 22......Page 656
Chapter 25......Page 658
Chapter 26......Page 661
Chapter 27......Page 663
Chapter 35......Page 667
Glossary......Page 670
A......Page 673
C......Page 674
F......Page 676
K......Page 677
M......Page 678
N......Page 679
P......Page 680
R......Page 681
S......Page 682
T......Page 683
Z......Page 684
Back Cover......Page 685