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Epidemiologic methods : studying the occurrence of illness PDF

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SECOND EDITION EPIDEMIOLOGIC METHODS Studying the Occurrence of Illness NOEL S. WEISS THOMAS D. KOEPSELL University of Washington 1 3 OxfordUniversityPressisadepartmentoftheUniversityofOxford. ItfurtherstheUniversity’sobjectiveofexcellenceinresearch,scholarship, andeducationbypublishingworldwide. Oxford NewYork Auckland CapeTown DaresSalaam HongKong Karachi KualaLumpur Madrid Melbourne MexicoCity Nairobi NewDelhi Shanghai Taipei Toronto Withofficesin Argentina Austria Brazil Chile CzechRepublic France Greece Guatemala Hungary Italy Japan Poland Portugal Singapore SouthKorea Switzerland Thailand Turkey Ukraine Vietnam OxfordisaregisteredtrademarkofOxfordUniversityPress intheUKandcertainothercountries. PublishedintheUnitedStatesofAmericaby OxfordUniversityPress 198MadisonAvenue,NewYork,NY10016 ©OxfordUniversityPress2014 Allrightsreserved.Nopartofthispublicationmaybereproduced,storedina retrievalsystem,ortransmitted,inanyformorbyanymeans,withouttheprior permissioninwritingofOxfordUniversityPress,orasexpresslypermittedbylaw, bylicense,orundertermsagreedwiththeappropriatereproductionrightsorganization. Inquiriesconcerningreproductionoutsidethescopeoftheaboveshouldbesenttothe RightsDepartment,OxfordUniversityPress,attheaddressabove. Youmustnotcirculatethisworkinanyotherform andyoumustimposethissameconditiononanyacquirer. LibraryofCongressCataloging-in-PublicationData Weiss,NoelS.,Koepsell,ThomasD.,author. Epidemiologicmethods:studyingtheoccurrenceofillness/NoelS.Weiss,ThomasD.Koepsell.—Secondedition. p.;cm. Includesbibliographicalreferences. ISBN978–0–19–531446–5(alk.paper) I. Weiss,NoelS.,1941–author. II. Title. III. Title:Epidemiologicmethods. [DNLM:1.EpidemiologicMethods.WA950] RA652.4 614.4—dc23 2014001643 1 3 5 7 9 8 6 4 2 PrintedintheUnitedStatesofAmerica onacid-freepaper EPIDEMIOLOGIC METHODS CONTENTS Preface vii 12. ConfoundingandItsControl: Advanced 225 1. Introduction:AnEpidemicofBlindnessin 13. RandomizedTrials 280 YoungChildren 1 14. CohortStudies 320 2. DiseasesandPopulations 10 15. Case-ControlStudies 340 3. DiseaseFrequency:Basic 27 16. EcologicalandMulti-LevelStudies 360 4. DiseaseFrequency:Advanced 48 17. InductionPeriodsandLatentPeriods 381 5. OverviewofStudyDesigns 76 18. ImprovingtheSensitivityof 6. SourcesofDataonDiseaseOccurrence 105 EpidemiologicStudies 390 7. Person,Place,andTime 128 19. Screening 407 8. InferringaCausalRelationBetween 20. OutbreakInvestigation 428 ExposureandDisease 156 21. EvaluatingtheEffectsof 9. MeasuresofExcessRisk 168 PoliciesonHealth 447 10. MeasurementError 179 Index 463 11. ConfoundingandItsControl:Basic 211 PREFACE Epidemiologyatanygiventimeissomethingmorethanthetotalofitsestablishedfacts.Itincludestheir orderlyarrangementintochainsofinferencewhichextendmoreorlessbeyondtheboundsofdirect observation.Suchofthesechainsasarewellandtrulylaidguideinvestigationtothefactsofthefuture. —WADEHAMPTONFROST1 Between us, we have accumulated more than 60 research themselves, or who need methodological person-yearsteachingcoursesontheprinciplesand expertise to interpret and synthesize properly the methods of epidemiology. We have jointly taught resultsofepidemiologic research producedbyoth- a two-course sequence at the University of Wash- ers. Itstartsatthebeginning, sotospeak, butcov- ington since the mid-1980s. During this time, we ersmuchofthematerialinmoredetailthanwould havelistenedtothereactionsofmanystudentswho be desired by a reader who wished only to have a werebeingintroducedtothisfield.Onecamefroma general appreciation of epidemiology. Those who womanwhosaidshederivedanalmostestheticplea- have already taken an introductory course in epi- sure from epidemiology: she found bothefficiency demiologymayfindhereawelcomereviewofbasic andbeautyintheprocessoftakingrawobservations concepts,aswellasnew,moreadvancedmaterial. ontheoccurrenceofanillnessinhumansandweav- A brief outline of the book may be helpful. ingtheminto“chainsofinferences”aboutthecauses Chapter1tellsthestoryofarealdiseaseoutbreak, ofthatillness,whichoftenhadthepotentialtolead introducing epidemiologic concepts and designs toprevention. alongthewaythatwillbecoveredindepthinlater We suspect that not too many of our students chapters.Chapter2setsforthanepidemiologicview wouldhaveusedthewordesthetictodescribeanyof of diseases and populations, leading into Chapters their feelings about our course, even their positive 3and4,onmeasuringdiseasefrequencyinpopula- feelings.(Somemighthavesaidthatanestheticbetter tions.Chapter5paintsthe“bigpicture”ofresearch characterizedsomeoftheclasssessions!) Nonethe- designs in epidemiology, so that their names are less,weliketothinkthatthefirststudenthadinter- familiarwhenusedinchaptersthatfollow.Chapters nalized what we were trying to convey—that the 6and7highlightseveralspecificsourcesofnumera- techniquesofstructuringobservationsandanalyzing toranddenominatordatainepidemiologyandpro- theinformationgathered(uponwhichsomuchtime videexamplesoftheperson/place/timeconceptual and effort are expended in class) are tobeused to frameworkthatunderliesmanydescriptiveepidemi- produce“...chains[that]arewellandtrulylaid...” ologicstudies. inthehopeofguiding“investigationtothefactsof As a transition into analytic epidemiology, the future.” Having produced extensive classroom Chapter8discussesthekindsofepidemiologicevi- materialstoachieveourgoals,wefeltthatwithalittle dencethatbearonaninferencethatanassociation (!)extralaborwecouldorganizethesematerialsinto may be causal. Chapter 9 also focuses on associ- abookforotherstudentsofepidemiology. ations, describing several quantitative measures of Whichstudents? Thebookisaimedatbeginning excess risk that can be calculated from epidemio- and intermediate students of epidemiologic meth- logic data. Chapter 10 introduces several quantita- ods. It is meant to serve as an introduction to the tivetechniquesforassessingthereliabilityandvalid- fieldforpeoplewhointendtoconductepidemiologic ity of epidemiologic measurements and describes 1Fromhisintroductionto:SnowJ.SnowonCholera. NewYork:TheCommonwealthFund,1936. viii PREFACE how measurement error affects observed associa- the applicability of the principles and methods to tions. Chapter 11 describes what confounding is, real-lifesituations. how it is traditionally assessed, and basic methods From the beginning, thebook hasbeen ajoint foritscontrol.Chapter12(newinthisedition)dis- enterprisebythetwoofus.Eachofusdraftedabout cussesmoreadvancedaspectsofconfounding:alter- halfofthechapters.Noelwasleadauthoronchapters native conceptualizations, causal diagrams, multi- 8–9,11,14–15,and17–18;Tomwasleadauthoron variableanalysis,data-reductionapproaches,inverse chapters1–7,12–13,16,and21.Chapters10and19 probability–weighting, instrumental variables, and wereco-authoredbybothofus.Eachofuswasthe time-dependentconfounding. primarycriticalrevieweroftheother’sdraftchapters, Chapters 13–16each focus in depth on a class andbothofusarepreparedtostandbythebookas of epidemiologic study designs: randomized trials, awhole.JenniferLloydandJeffreyDuchin,epidemi- cohortstudies,case-controlstudies,andecologicand ologistswithPublicHealthSeattleandKingCounty, multi-levelstudies.Chapter17discussestheimplica- contributed the chapter on outbreak investigation, tionsforepidemiologicstudiesofthetemporalrela- forwhichwearegrateful. tionshipbetweenexposureandoutcome.Chapter18 Therebeingnobetterwayofdecidingtheorder describeswaysinwhichepidemiologicstudiescanbe ofournamesontheentirebook,weagreedtoalter- designedandanalyzedtoenhancethelikelihoodthat natetheorder witheachedition. Astheresultofa atrueassociationisdetected. fateful coin flip, Tom was first author on the first Chapters19–21covermethodologicalaspectsof edition, andNoelisnowfirstauthoronthesecond severalkeytopicsinepidemiology:screeningfordis- edition. ease, short-term disease outbreaks, and evaluating Wegratefullyacknowledgehelpfulcommentson the effects of societal and institutional policies on draftchaptersfromseveralcolleaguesandstudents. health. AliRowhani-Rahbar,PeterCummings,andAmanda As the book’s title indicates, the discussion Phipps kindly reviewed some of the chapters and throughoutfocusesonthemeansbywhichepidemi- made many useful suggestions. Several cohorts of ologicresearchisconductedandinterpreted,noton studentsinourtwo-quartercoursesequenceonepi- thesubstantiveknowledgeaboutspecificdiseasesor demiologic methods have used the first edition as exposuresthathasaccumulatedfromspecificstudies. their main textbook and provided feedback about Twothingsthatwilldistinguishthisbookfrom errorsandpointsofconfusion. Thesecondedition otherswithsimilargoalsareitsliberaluseofexam- hasbenefitedfromtheirsharpeyes. plesfrom thepublishedmedical literature, andthe sets of questions that appear at the end of most NoelS.Weiss chapters. Most ofthesequestionspertain toactual ThomasD.Koepsell publishedstudies, ourpurposebeing toemphasize 1 Introduction: An Epidemic of Blindness in Young Children DISCOVERY OnFebruary14,1941,aBostonpediatriciannamed Dr.StewartCliffordwasmakingaroutinehousecall ononeofhispatients, ababygirlthenaboutthree monthsold.Shehadbeenbornprematurely,weigh- ingaboutfourpoundsatbirth.Butexceptforsome brief early episodes ofturningblue(cyanosis), she haddonewellduringherhospitalstayandhadbeen discharged. AsDr.Cliffordexaminedher,henoticedsome- thingoddaboutthebaby’seyes.Hergazewandered aimlessly, and her eyes jerked rapidly from side to side. There were prominent gray opacities in both eyes.Hewaspuzzledandconcerned:byallappear- ances,thebabygirlwasblind. Dr.CliffordcalledinDr.PaulChandler,aleading Bostonophthalmologist.Dr.Chandlerhadnotseen thisbaby’sconditionbefore.Topermitafullerevalu- FIGURE 1.1 Cross-sectionofaneyeshowingretro- ation,thebabywashospitalizedagainandexamined lentalfibroplasia. underanesthesia.Themainabnormality(illustrated (ReprintedcourtesyofDr.ArnallPatz) inFigure1.1)wasagraymassofscartissueattached tothebackofthelensandcoveredwithbloodves- fibroplasia(RLF),referringtoproliferationoffibrous sels.Itlaterbecameclearthatthistissuewasallthat scartissuebehindthelens. remainedofaretinathathadbeensobadlydamaged byhemorrhageandinflammationthatithaddelam- inatedfromthebackoftheeye,becomescarredand EPIDEMIC fibrous,andfloatedforwardtoaffixitselftothelens. Once Dr. Terry’s description of RLF appeared in Thebabygirlwascompletelyandirreversiblyblind. the medical literature, other physicians began to Thisfirstcaseprovednottobeabizarre,isolated look for it and to find it. By1945, Dr. Terry pub- occurrence.Withinaweek,Dr.Cliffordencountered lishedadescriptionof117cases,allbutfiveofthem anotherblindinfantwiththesamecondition.Soon in babies who had been born prematurely (Terry, another consultantophthalmologist, Dr. Theodore 1945). Across the country, the California School Terry, hadgatheredinformation onfivesuchcases fortheBlindreportedasharpriseinthenumberof in the Boston area, whom he described in an arti- RLF cases over time among babies born in South- cleintheAmericanJournalofOphthalmology(Terry, ern California, as shown in Figure 1.2 (Silverman, 1942). Shared pathological features among these 1980).Withinjustafewyearsafteritsdiscovery,RLF early cases led to calling the condition retrolental wentfrombeingliterallyunknowntobeingthemost

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