Skip to main content
SpringerLink
Log in

Case-Control Studies

  • Chapter
Handbook of Epidemiology

Abstract

The case-control study examines the association between disease and potential risk factors by taking separate samples of diseased cases and of controls at risk of developing disease. Information may be collected for both cases and controls on genetic, social, behavioral, environmental or other determinants of disease risk. The basic study design has a long history, extending back at least to Guy’s 1843 comparison of the occupations of men with pulmonary consumption to the occupations of men having other diseases (Lilienfeld and Lilienfeld 1979). Beginning in the 1920’s, it was used to link cancer to environmental and hormonal exposures. Broders (1920) discovered an association between pipe smoking and lip cancer; Lane-Claypon (1926), who selected matched hospital controls, investigated the relationship between reproductive experience and female breast cancer; and Lombard and Doering (1928) related pipe smoking to oral cancer. The publication in 1950 of three reports on the association between cigarette smoking and lung cancer generated enormous interest in case-control methodology as well as bitter criticism (Levin et al. 1950; Wynder and Graham 1950; Doll and Hill 1950). The landmark study of Doll and Hill (1950, 1952), in particular, inspired future generations of epidemiologists to use this methodology. It remains to this day a model for the design and conduct of case-control studies, with excellent suggestions on how to reduce or eliminate selection, interview and recall bias.

This is a preview of subscription content, log in via an institution to check access.

Access this chapter

Log in via an institution
Chapter
USD 29.95
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
eBook
USD 199.00
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever

Tax calculation will be finalised at checkout

Purchases are for personal use only

Institutional subscriptions

Preview

Unable to display preview. Download preview PDF.

Unable to display preview. Download preview PDF.

References

  • Aird L, Bentall HH, Roberts JAF (1953) A relationship between cancer of stomach and the ABO blood groups. British Medical Journal 1:799–801

    Google Scholar 

  • Andrieu N, Goldstein AM (1998) Epidemiologic and genetic approaches in the study of gene-environment interaction: An overview of available methods. Epidemiologic Reviews 20:137–147

    Google Scholar 

  • Armenian HK, Lilienfeld DE (1994) Overview and historical perspective. Epidemiologic Reviews 16:1–5

    Google Scholar 

  • Armstrong RW, Armstrong MJ, Yu MC, Henderson BE (1983) Salted fish and inhalants as risk-factors for nasopharyngeal carcinoma in Malaysian Chinese. Cancer Research 43:2967–2970

    Google Scholar 

  • Austin H, Hill HA, Flanders WD, Greenberg RS (1994) Limitations in the application of case-control methodology. Epidemiologic Reviews 16:65–76

    Google Scholar 

  • Barlow WE (1994) Robust variance estimation for the case-cohort design. Biometrics 50:1064–1072

    Article  MATH  Google Scholar 

  • Benichou J, Gail MH (1995) Methods of inference for estimates of absolute risk derived from population-based case-control studies. Biometrics 51:182–194

    Article  MATH  Google Scholar 

  • Berkson J (1946) Limitations of the application of fourfold table analysis to hospital data. Biometrics Bulletin 2:47–53

    Article  Google Scholar 

  • Breslow N (1982) Design and analysis of case-control studies. Annual Review of Public Health 3:29–54

    Article  Google Scholar 

  • Breslow NE (1996) Statistics in epidemiology: The case-control study. Journal of the American Statistical Association 91:14–28

    Article  MATH  MathSciNet  Google Scholar 

  • Breslow NE (2003) Are statistical contributions to medicine undervalued? Biometrics 59:1–8

    Article  MathSciNet  Google Scholar 

  • Breslow NE, Cain KC (1988) Logistic regression for two-stage case-control data. Biometrika 75:11–20

    Article  MATH  MathSciNet  Google Scholar 

  • Breslow NE, Chatterjee N (1999) Design and analysis of two-phase studies with binary outcomes applied to Wilms tumor prognosis. Applied Statistics 48:457–468

    MATH  Google Scholar 

  • Breslow NE, Day NE (1980) Statistical methods in cancer research I: The analysis of case-control studies. International Agency for Research on Cancer, Lyon

    Google Scholar 

  • Breslow NE, Lubin JH, Marek P, Langholz B (1983) Multiplicative models and cohort analysis. Journal of the American Statistical Association 78:1–12

    Article  MATH  Google Scholar 

  • Broders AC (1920) Squamous-cell epithelioma of the lip. A study of five hundred and thirty-seven cases. Journal of the American Medical Association 74:656–664

    Google Scholar 

  • Carroll RJ, Ruppert D, Stefanski LA(1995)Measurement error in nonlinear models. Chapman and Hall, London

    MATH  Google Scholar 

  • Chase G, Klauber MR (1965) A graph of sample sizes for retrospective studies. American Journal of Public Health 55:1993–1996

    Google Scholar 

  • Cole P (1979) The evolving case-control study. Journal of Chronic Disease 32:15–27

    Article  Google Scholar 

  • Comstock GW (1994) Evaluating vaccination effectiveness and vaccine efficacy by means of case-control studies. Epidemiologic Reviews 16:77–89

    Google Scholar 

  • Cornfield J (1951) A method of estimating comparative rates from clinical data. Applications to cancer of the lung, breast, and cervix. Journal of the National Cancer Institute 11:1269–1275

    Google Scholar 

  • Correa A, Stewart WF, Yeh HC, Santos-Burgoa C (1994) Exposure measurement in case-control studies: Reported methods and recommendations. Epidemiologic Reviews 16:18–32

    Google Scholar 

  • Cox DR (1972) Regression models and life-tables (with discussion). Journal of the Royal Statistical Society (Series B) 34:187–220

    MATH  Google Scholar 

  • Daling JR, Weiss NS, Metch BJ, Chow WH, Soderstrom RM, Stadel BV (1985) Primary tubal infertility in relation to the use of an intrauterine-device. New England Journal of Medicine 312:937–941

    Article  Google Scholar 

  • Doll R, Hill AB (1950) Smoking and carcinoma of the lung. Preliminary report. British Medical Journal 2:739–748

    Google Scholar 

  • Doll R, Hill AB (1952) A study of the aetiology of carcinoma of the lung. British Medical Journal 2:1271–1286

    Article  Google Scholar 

  • Dorn HF (1959) Some problems arising in prospective and retrospective studies of the etiology of disease. New England Journal of Medicine 261:571–579

    Article  Google Scholar 

  • Fleming PJ, Gilbert R, Azaz Y, Berry PJ, Rudd PT, Stewart A, Hall E (1990) Interaction between bedding and sleeping position in the sudden-infant-death-syndrome — a population based case-control study. British Medical Journal 301:85–89

    Google Scholar 

  • Gordis L (1982) Should dead cases be matched to dead controls? American Journal of Epidemiology 115:1–5

    Google Scholar 

  • Graubard BI, Fears TR, Gail MH (1989) Effects of cluster sampling on epidemiologic analysis in population-based case-control studies (Corr: V47 p. 779–780). Biometrics 45:1053–1071

    Article  MATH  Google Scholar 

  • Greenberg ER (1990) Random digit dialing for control selection — A review and a caution on its use in studies of childhood-cancer. American Journal of Epidemiology 131:1–5

    Google Scholar 

  • Greenland S (1987) Estimation of exposure-specific rates from sparse case-control data. Journal of Chronic Disease 40:1087–1094

    Article  Google Scholar 

  • Greenland S, Robins JM (1985) Confounding and misclassification. American Journal of Epidemiology 122:495–506

    Google Scholar 

  • Greenland S, Thomas DC (1982) On the need for the rare disease assumption in case-control studies. American Journal of Epidemiology 116:547–553

    Google Scholar 

  • Harlow BL, Davis S (1988) Two one-step methods for household screening and interviewing using random digit dialing. American Journal of Epidemiology 127:857–863

    Google Scholar 

  • Henderson MM, Kushi LH, Thompson DJ, Gorbach SL, Clifford CK, Thompson RS (1990) Feasibility of a randomized trial of a low-fat diet for the prevention of breast-cancer — Dietary compliance in the womens health trial vanguard study. Preventive Medicine 19:115–133

    Article  Google Scholar 

  • Herbst AL, Ulfelder H, Poskanzer DC (1971) Adenocarcinoma of the vagina. New England Journal of Medicine 284:878–881

    Article  Google Scholar 

  • Hill AB (1953) Observation and experiment. New England Journal of Medicine 248:995–1001

    Article  Google Scholar 

  • Hill AB (1965) The environment and disease: association or causation? Proceedings of the Royal Statistical Society of Medicine 58:295–300

    Google Scholar 

  • Hill AB (1971) Principles of medical statistics. Oxford University Press, New York

    Google Scholar 

  • Horvitz DG, Thompson DJ (1952) A generalization of sampling without replacement from a finite universe. Journal of the American Statistical Association 47:663–685

    Article  MATH  MathSciNet  Google Scholar 

  • Hsieh DA, Manski CF, McFadden D (1985) Estimation of response probabilities from augmented retrospective observations. Journal of the American Statistical Association 80:651–662

    Article  MATH  Google Scholar 

  • Ibrahim MA, Spitzer WO (1979) The case-control study: The problem and the prospect. Journal of Chronic Disease 32:139–144

    Article  Google Scholar 

  • Jablon S, Neel JV, Gershowitz H, Atkinson GF (1967) The NAS-NRC twin panel: Methods of construction of the panel, zygosity diagnosis, and proposed use. American Journal of Human Genetics 19:133–161

    Google Scholar 

  • Kelsey JL, Whittemore AS, Evans AS, Thompson WD (1996) Methods in observational epidemiology, 2nd edn. Oxford University Press, New York

    Google Scholar 

  • Khoury MJ, Beaty TH (1994) Applications of the case-control method in genetic epidemiology. Epidemiologic Reviews 16:134–150

    Google Scholar 

  • Kupper LL, McMichael AJ, Spirtas R (1975) A hybrid epidemiologic study design useful in estimating relative risk. Journalof the American Statistical Association 70:524–528

    Article  Google Scholar 

  • Lane-Claypon JE (1926) A further report on cancer of the breast. Her Majesty’s Stationery Office, London

    Google Scholar 

  • Langholz B, Borgan O (1995) Counter-matching: A stratified nested case-control sampling method. Biometrika 82:69–79

    Article  MATH  Google Scholar 

  • Langholz B, Borgan O (1997) Estimation of absolute risk from nested case-control data. Biometrics 53:767–774

    Article  MATH  Google Scholar 

  • Langholz B, Goldstein L (1996) Risk set sampling in epidemiologic cohort studies. Statistical Science 11:35–53

    Article  Google Scholar 

  • Levin ML, Goldstein H, Gerhardt PR (1950) Cancer and tobacco smoking. A preliminary report. Journal of the American Medical Association 143:336–338

    Google Scholar 

  • Liddell FDK, McDonald JC, Thomas DC (1977) Methods of cohort analysis: Appraisal by application to asbestos mining. Journal of the Royal Statistical Society (Series A) 140:469–491

    Google Scholar 

  • Lilienfeld AM, Lilienfeld DE (1979) A century of case-control studies: Progress? Journal of Chronic Disease 32:5–13

    Article  Google Scholar 

  • Lin DY, Ying Z (1993) Cox regression with incomplete covariate measurements. Journal of the American Statistical Association 88:1341–1349

    Article  MATH  MathSciNet  Google Scholar 

  • Little RJA, Rubin DB (2002) Statistical analysis with missing data, 2nd edn. Wiley, New York

    MATH  Google Scholar 

  • Lombard HL, Doering CR (1928) Cancer studies in Massachusetts. 2. Habits, characteristics and environment of individuals with and without cancer. New England Journal of Medicine 198:481–487

    Article  Google Scholar 

  • MacMahon B, Cole P, Lin TM, Lowe CR, Mirra AP, Ravnihar B, Salber EJ, Valaoras VG, Yuasa S (1970) Age at first birth and breast cancer risk. Bulletin of the World Health Organization 43:209–221

    Google Scholar 

  • Mantel N, Haenszel W (1959) Statistical aspects of the analysis of data from retrospective studies of disease. Journal of the National Cancer Institute 22:719–748

    Google Scholar 

  • Miettinen O (1982) Design options in epidemiologic research: An update. Scandinavian Journal of Work Environment and Health 8:7–14

    Google Scholar 

  • Miettinen OS (1970) Matching and design efficiency in retrospective studies. American Journal of Epidemiology 91:111–118

    Google Scholar 

  • Miettinen O (1976) Estimability and estimation in case-referent studies. American Journal of Epidemiology 103:226–235

    Google Scholar 

  • Miettinen OS (1985) Theoretical epidemiology: Principles of occurrence research in medicine. Wiley, New York

    Google Scholar 

  • Neutra RR, Drolette ME (1978) Estimating exposure-specific disease rates from case-control studies using Bayes theorem. American Journal of Epidemiology 108:214–222

    Google Scholar 

  • Neyman J (1955) Statistics — servant of all sciences. Science 122:401–406

    Article  Google Scholar 

  • O’Neil MJ (1979) Estimating the nonresponse bias due to refusals in telephone surveys. Public Opinion Quarterly 43:218–232

    Article  Google Scholar 

  • Poole C (1987) Critical appraisal of the exposure-potential restriction rule. American Journal of Epidemiology 125:179–183

    Google Scholar 

  • Prentice RL (1986) A case-cohort design for epidemiologic cohort studies and disease prevention trials. Biometrika 73:1–11

    Article  MATH  MathSciNet  Google Scholar 

  • Prentice RL (1996) Measurement error and results from analytic epidemiology: dietary fat and breast cancer. Journal of the National Cancer Institute 88:1738–1747

    Article  Google Scholar 

  • Prince AM, Szmuness W, Michon J, Demaille J, Diebolt G, Linhard J, Quenum C, Sankale M (1975) A case-control study of the association between primary liver cancer and hepatitis B infection in Senegal. International Journal of Cancer 16:376–383

    Article  Google Scholar 

  • Robins J, Pike M (1990) The validity of case-control studies with nonrandom selection of controls. Epidemiology 1:273–284

    Article  Google Scholar 

  • Robins JM, Gail MH, Lubin JH (1986) More on ‘biased selection of controls for case-control analyses of cohort studies’. Biometrics 42:293–29

    Article  Google Scholar 

  • Robison LL, Daigle A (1984) Control selection using random digit dialing for cases of childhood cancer. American Journal of Epidemiology 120:164–165

    Google Scholar 

  • Rodrigues L, Kirkwood BR (1990) Case-control designs in the study of common diseases: Updates on the demise of the rare disease assumption and the choice of sampling scheme for controls. International Journal of Epidemiology 19:205–213

    Article  Google Scholar 

  • Rosenbaum PR (1987) The role of a second control group in an observational study (with discussion). Statistical Science 2:292–316

    Google Scholar 

  • Rothman KJ (1986) Modern epidemiology. Little, Brown, Boston

    Google Scholar 

  • Rothman KJ, Greenland S (1998) Modern epidemiology, 2nd edn. Lippincott-Raven, Philadelphia

    Google Scholar 

  • Schlesselman JJ (1982) Case-control studies. Oxford University Press, New York

    Google Scholar 

  • Schlesselman JJ, Stadel BV (1987) Exposure opportunity in epidemiologic studies. American Journal of Epidemiology 125:174–178

    Google Scholar 

  • Sheehe PR (1962) Dynamic risk analysis in retrospective matched pair studies of disease. Biometrics 18:323–341

    Article  Google Scholar 

  • Smith DC, Prentice R, Thompson DJ, Herrmann W (1975) Association of exogenous estrogen and endometrial carcinoma. New England Journal of Medicine 293:1164–1167

    Article  Google Scholar 

  • Smith PG, Day NE (1984) The design of case-control studies: The influence of confounding and interaction effects. International Journal of Epidemiology 13:356–365

    Article  Google Scholar 

  • Smith PG, Rodrigues LC, Fine PEM (1984) Assessment of the protective efficacy of vaccines against common diseases using case-control and cohort studies. International Journal of Epidemiology 13:87–93

    Article  Google Scholar 

  • Taubes G (1995) Epidemiology faces its limits. Science 269:164–169

    Article  MathSciNet  Google Scholar 

  • Thomas DC, Greenland S (1983) The relative efficiencies of matched and independent sample designs for case-control studies. Journal of Chronic Disease 36:685–697

    Article  Google Scholar 

  • Tuyns AJ, Péquignot G, Jensen OM (1977) Le cancer de l’oesophage en Ille-et-Vilaine en fonction des niveaux de consommation d’alcool et de tabac. Bulletin du Cancer 64:45–60

    Google Scholar 

  • Wacholder S (1995) Design issues in case-control studies. Statistical Methods in Medical Research 4:293–309

    Google Scholar 

  • Wacholder S, McLaughlin JK, Silverman DT, Mandel JS (1992) Selection of controls in case-control studies I. Principles. American Journal of Epidemiology 135:1019–1028

    Google Scholar 

  • Wacholder S, Silverman DT, McLaughlin JK, Mandel JS (1992) Selection of controls in case-control studies II. Types of controls. American Journal of Epidemiology 135:1029–1041

    Google Scholar 

  • Waksberg J (1978) Sampling methods for random digit dialing. Journal of the American Statistical Association 73:40–46

    Article  Google Scholar 

  • Weinberg CR, Wilcox AJ (1998) Reproductive epidemiology. In: Rothman KJ, Greenland S (eds) Modern epidemiology, 2nd edn., Chap. 29, pp 585–608. Lippincott-Raven, Philadeplphia

    Google Scholar 

  • Weiss NS (1994) Application of the case-control method in the evaluation of screening. Epidemiologic Reviews 16:102–108

    Google Scholar 

  • Weiss NS (2002) Can the’ specificity’ of an association be rehabilitated as a basis for supporting a causal hypothesis? Epidemiology 13:6–8

    Article  Google Scholar 

  • White JE (1982) A two stage design for the study of the relationship between a rare exposure and a rare disease. American Journal of Epidemiology 115:119–128

    Google Scholar 

  • Wynder EL, Graham EA (1950) Tobacco smoking as a possible etiologic factor in bronchogenic carcinoma. A study of six hundred and eighty-four proved cases. Journal of the American Medical Association 143:329–336

    Google Scholar 

  • Yu MC, Ho JHC, Lai SH, Henderson BE (1986) Cantonese-style salted fish as a cause of nasopharyngeal carcinoma-Report of a case-control study in Hong-Kong. Cancer Research 46:956–961

    Google Scholar 

  • Ziel HK, Finkle WD (1975) Increased risk of endometrial carcinoma among users of conjugated estrogens. New England Journal of Medicine 293:1167–1170

    Article  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Department of Biostatistics, University of Washington, Box 357232, Seattle, WA, 98195-7232, USA

    Norman E. Breslow

Authors
  1. Norman E. Breslow
    View author publications

    You can also search for this author in PubMed Google Scholar

Editor information

Editors and Affiliations

  1. Division of Epidemiological Methods and Ethiologic Research, Bremen Institute for Prevention Research and Social Medicine (BIPS), Linzer Str. 10, 28359, Bremen, Germany

    Wolfgang Ahrens  & Iris Pigeot  & 

  2. Division of Biometrie and Data Management, Bremen Institute for Prevention Research and Social Medicine (BIPS), Linzer Str. 10, 28359, Bremen, Germany

    Wolfgang Ahrens  & Iris Pigeot  & 

Rights and permissions

Reprints and permissions

Copyright information

© 2005 Springer-Verlag Berlin Heidelberg

About this chapter

Cite this chapter

Breslow, N.E. (2005). Case-Control Studies. In: Ahrens, W., Pigeot, I. (eds) Handbook of Epidemiology. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-540-26577-1_7

Download citation

Publish with us

Policies and ethics

Search

Navigation