Handbook of Statistics 27: Epidemiology and Medical Statistics

871 Pages·2007·4.01 MB·English

by  C.R. Rao

HANDBOOK OF STATISTICS
VOLUME 27
Handbook of Statistics
VOLUME 27
General Editor
C.R. Rao
Amsterdam (cid:2) Boston (cid:2)Heidelberg (cid:2) London(cid:2) New York(cid:2) Oxford
Paris (cid:2) San Diego(cid:2) SanFrancisco (cid:2) Singapore (cid:2)Sydney(cid:2) Tokyo
Epidemiology and Medical Statistics
Edited by
C.R. Rao
Center forMultivariate Analysis
Departmentof Statistics, ThePennsylvaniaState University
UniversityPark, PA, USA
J.P. Miller
Division of Biostatistics
School of Medicine, Washington Universityin St. Louis
St.Louis, MO, USA
D.C. Rao
Division of Biostatistics
School of Medicine, Washington Universityin St. Louis
St.Louis, MO, USA
Amsterdam(cid:2)Boston(cid:2)Heidelberg(cid:2)London(cid:2)NewYork(cid:2)Oxford
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Table of contents
Preface xiii
Contributors xv
Ch. 1. Statistical Methods and Challenges in Epidemiology and
Biomedical Research 1
Ross L. Prentice
1.Introduction 1
2.Characterizingthestudycohort 3
3.Observationalstudymethodsandchallenges 6
4.Randomizedcontrolledtrials 12
5.Intermediate,surrogate,andauxiliaryoutcomes 17
6.Multipletestingissuesandhigh-dimensionalbiomarkers 18
7.FurtherdiscussionandtheWomen’sHealthInitiativeexample 21
References 22
Ch. 2. Statistical Inference for Causal Effects, With Emphasis on
Applications in Epidemiology and Medical Statistics 28
Donald B. Rubin
1.Causalinferenceprimitives 28
2.Theassignmentmechanism 36
3.Assignment-basedmodesofcausalinference 41
4.Posteriorpredictivecausalinference 47
5.Complications 55
References 58
Ch. 3. Epidemiologic Study Designs 64
Kenneth J. Rothman, Sander Greenland and Timothy L. Lash
1.Introduction 64
2.Experimentalstudies 65
3.Nonexperimentalstudies 73
4.Cohortstudies 73
v
vi Tableofcontents
5.Case-controlstudies 84
6.Variantsofthecase-controldesign 97
7.Conclusion 104
References 104
Ch. 4. Statistical Methods for Assessing Biomarkers and Analyzing
Biomarker Data 109
Stephen W. Looney and Joseph L. Hagan
1.Introduction 109
2.Statisticalmethodsforassessingbiomarkers 110
3.Statisticalmethodsforanalyzingbiomarkerdata 126
4.Concludingremarks 143
References 144
Ch. 5. Linear and Non-Linear Regression Methods in Epidemiology and
Biostatistics 148
Eric Vittinghoff, Charles E. McCulloch, David V. Glidden and Stephen
C. Shiboski
1.Introduction 148
2.Linearmodels 151
3.Non-linearmodels 167
4.Specialtopics 176
References 182
Ch. 6. Logistic Regression 187
Edward L. Spitznagel Jr.
1.Introduction 187
2.Estimationofasimplelogisticregressionmodel 188
3.Twomeasuresofmodelfit 191
4.Multiplelogisticregression 192
5.Testingforinteraction 194
6.Testinggoodnessoffit:Twomeasuresforlackoffit 195
7.Exactlogisticregression 196
8.Ordinallogisticregression 201
9.Multinomiallogisticregression 204
10.Probitregression 206
11.Logisticregressionincase–controlstudies 207
References 209
Ch. 7. Count Response Regression Models 210
Joseph M. Hilbe and William H. Greene
1.Introduction 210
2.ThePoissonregressionmodel 212
3.Heterogeneityandoverdispersion 224
4.Importantextensionsofthemodelsforcounts 230
Tableofcontents vii
5.Software 247
6.Summaryandconclusions 250
References 251
Ch. 8. Mixed Models 253
Matthew J. Gurka and Lloyd J. Edwards
1.Introduction 253
2.Estimationforthelinearmixedmodel 259
3.Inferenceforthemixedmodel 261
4.Selectingthebestmixedmodel 264
5.Diagnosticsforthemixedmodel 268
6.Outliers 270
7.Missingdata 270
8.Powerandsamplesize 272
9.Generalizedlinearmixedmodels 273
10.Nonlinearmixedmodels 274
11.Mixedmodelsforsurvivaldata 275
12.Software 276
13.Conclusions 276
References 277
Ch. 9. Survival Analysis 281
John P. Klein and Mei-Jie Zhang
1.Introduction 281
2.Univariateanalysis 282
3.Hypothesistesting 288
4.Regressionmodels 295
5.Regressionmodelsforcompetingrisks 310
References 317
Ch. 10. A Review of Statistical Analyses for Competing Risks 321
Melvin L. Moeschberger, Kevin P. Tordoff and Nidhi Kochar
1.Introduction 321
2.Approachestothestatisticalanalysisofcompetingrisks 324
3.Example 327
4.Conclusion 339
References 340
Ch. 11. Cluster Analysis 342
William D. Shannon
1.Introduction 342
2.Proximitymeasures 344
3.Hierarchicalclustering 350
4.Partitioning 355
5.Ordination(scaling) 358
viii Tableofcontents
6.Howmanyclusters? 361
7.Applicationsinmedicine 364
8.Conclusion 364
References 365
Ch. 12. Factor Analysis and Related Methods 367
Carol M. Woods and Michael C. Edwards
1.Introduction 367
2.Exploratoryfactoranalysis(EFA) 368
3.Principlecomponentsanalysis(PCA) 375
4.Confirmatoryfactoranalysis(CFA) 375
5.FAwithnon-normalcontinuousvariables 379
6.FAwithcategoricalvariables 380
7.SamplesizeinFA 382
8.ExamplesofEFAandCFA 383
9.Additionalresources 389
AppendixA 391
AppendixB 391
References 391
Ch. 13. Structural Equation Modeling 395
Kentaro Hayashi, Peter M. Bentler and Ke-Hai Yuan
1.Modelsandidentification 395
2.Estimationandevaluation 399
3.ExtensionsofSEM 410
4.Somepracticalissues 415
References 418
Ch. 14. Statistical Modeling in Biomedical Research: Longitudinal
Data Analysis 429
Chengjie Xiong, Kejun Zhu, Kai Yu and J. Philip Miller
1.Introduction 429
2.Analysisoflongitudinaldata 431
3.Designissuesofalongitudinalstudy 456
References 460
Ch. 15. Design and Analysis of Cross-Over Trials 464
Michael G. Kenward and Byron Jones
1.Introduction 464
2.Thetwo-periodtwo-treatmentcross-overtrial 467
3.Higher-orderdesigns 476
4.Analysiswithnon-normaldata 482
5.Otherapplicationareas 485
6.Computersoftware 488
References 489
Tableofcontents ix
Ch. 16. Sequential and Group Sequential Designs in Clinical Trials:
Guidelines for Practitioners 491
Madhu Mazumdar and Heejung Bang
1.Introduction 492
2.Historicalbackgroundofsequentialprocedures 493
3.Groupsequentialproceduresforrandomizedtrials 494
4.StepsforGSDdesignandanalysis 507
5.Discussion 508
References 509
Ch. 17. Early Phase Clinical Trials: Phases I and II 513
Feng Gao, Kathryn Trinkaus and J. Philip Miller
1.Introduction 513
2.PhaseIdesigns 514
3.PhaseIIdesigns 526
4.Summary 539
References 541
Ch. 18. Definitive Phase III and Phase IV Clinical Trials 546
Barry R. Davis and Sarah Baraniuk
1.Introduction 546
2.Questions 548
3.Randomization 550
4.Recruitment 551
5.Adherence/samplesize/power 552
6.Dataanalysis 554
7.Dataqualityandcontrol/datamanagement 558
8.Datamonitoring 558
9.PhaseIVtrials 563
10.Dissemination–trialreportingandbeyond 564
11.Conclusions 565
References 565
Ch. 19. Incomplete Data in Epidemiology and Medical Statistics 569
Susanne Ra¨ssler, Donald B. Rubin and Elizabeth R. Zell
1.Introduction 569
2.Missing-datamechanismsandignorability 571
3.Simpleapproachestohandlingmissingdata 573
4.Singleimputation 574
5.Multipleimputation 578
6.Directanalysisusingmodel-basedprocedures 581
7.Examples 584
8.Literaturereviewforepidemiologyandmedicalstudies 586
9.Summaryanddiscussion 587
AppendixA 588
AppendixB 592
References 598