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 1920s, 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.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
References
Aird L, Bentall HH, Roberts JAF (1953) A relationship between cancer of stomach and the ABO blood groups. Br Med J 1:799–801
Andrieu N, Goldstein AM (1998) Epidemiologic and genetic approaches in the study of gene-environment interaction: an overview of available methods. Epidemiol Rev 20:137–147
Armenian HK, Lilienfeld DE (1994) Overview and historical perspective. Epidemiol Rev 16:1–5
Armstrong RW, Armstrong MJ, Yu MC, Henderson BE (1983) Salted fish and inhalants as risk-factors for nasopharyngeal carcinoma in Malaysian Chinese. Cancer Res 43:2967–2970
Austin H, Hill HA, Flanders WD, Greenberg RS (1994) Limitations in the application of case-control methodology. Epidemiol Rev 16:65–76
Barlow WE (1994) Robust variance estimation for the case-cohort design. Biometrics 50: 1064–1072
Benichou J, Gail MH (1995) Methods of inference for estimates of absolute risk derived from population-based case-control studies. Biometrics 51:182–194
Berkson J (1946) Limitations of the application of fourfold table analysis to hospital data. Biom Bull 2:47–53
Breslow N (1982) Design and analysis of case-control studies. Annu Rev Public Health 3:29–54
Breslow NE (1996) Statistics in epidemiology: the case-control study. J Am Stat Assoc 91:14–28
Breslow NE (2003) Are statistical contributions to medicine undervalued? Biometrics 59:1–8
Breslow NE, Cain KC (1988) Logistic regression for two-stage case-control data. Biometrika 75:11–20
Breslow NE, Chatterjee N (1999) Design and analysis of two-phase studies with binary outcomes applied to Wilms tumor prognosis. Appl Stat 48:457–468
Breslow NE, Day NE (1980) Statistical methods in cancer research I: the analysis of case-control studies. International Agency for Research on Cancer, Lyon
Breslow NE, Lubin JH, Marek P, Langholz B (1983) Multiplicative models and cohort analysis. J Am Stat Assoc 78:1–12
Broders AC (1920) Squamous-cell epithelioma of the lip. A study of five hundred and thirty-seven cases. J Am Med Assoc 74:656–664
Carroll RJ, Ruppert D, Stefanski LA (1995) Measurement error in nonlinear models. Chapman and Hall, London
Chase G, Klauber MR (1965) A graph of sample sizes for retrospective studies. Am J Public Health 55:1993–1996
Cole P (1979) The evolving case-control study. J Chronic Dis 32:15–27
Comstock GW (1994) Evaluating vaccination effectiveness and vaccine efficacy by means of case-control studies. Epidemiol Rev 16:77–89
Cornfield J (1951) A method of estimating comparative rates from clinical data. Applications to cancer of the lung, breast, and cervix. J Natl Cancer Inst 11:1269–1275
Correa A, Stewart WF, Yeh HC, Santos-Burgoa C (1994) Exposure measurement in case-control studies: reported methods and recommendations. Epidemiol Rev 16:18–32
Cox DR (1972) Regression models and life-tables (with discussion). J R Stat Soc (Ser B) 34: 187–220
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. N Engl J Med 312:937–941
Doll R, Hill AB (1950) Smoking and carcinoma of the lung. Preliminary report. Br Med J 2:739–748
Doll R, Hill AB (1952) A study of the aetiology of carcinoma of the lung. Br Med J 2:1271–1286
Dorn HF (1959) Some problems arising in prospective and retrospective studies of the etiology of disease. N Engl J Med 261:571–579
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. Br Med J 301:85–89
Gordis L (1982) Should dead cases be matched to dead controls? Am J Epidemiol 115:1–5
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
Greenberg ER (1990) Random digit dialing for control selection – a review and a caution on its use in studies of childhood-cancer. Am J Epidemiol 131:1–5
Greenland S (1987) Estimation of exposure-specific rates from sparse case-control data. J Chronic Dis 40:1087–1094
Greenland S, Robins JM (1985) Confounding and misclassification. Am J Epidemiol 122:495–506
Greenland S, Thomas DC (1982) On the need for the rare disease assumption in case-control studies. Am J Epidemiol 116:547–553
Harlow BL, Davis S (1988) Two one-step methods for household screening and interviewing using random digit dialing. Am J Epidemiol 127:857–863
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. Prev Med 19:115–133
Herbst AL, Ulfelder H, Poskanzer DC (1971) Adenocarcinoma of the vagina. N Engl J Med 284:878–881
Hill AB (1953) Observation and experiment. N Engl J Med 248:995–1001
Hill AB (1965) The environment and disease: association or causation? Proc R Stat Soc Med 58:295–300
Hill AB (1971) Principles of medical statistics. Oxford University Press, New York
Horvitz DG, Thompson DJ (1952) A generalization of sampling without replacement from a finite universe. J Am Stat Assoc 47:663–685
Hsieh DA, Manski CF, McFadden D (1985) Estimation of response probabilities from augmented retrospective observations. J Am Stat Assoc 80:651–662
Ibrahim MA, Spitzer WO (1979) The case-control study: the problem and the prospect. J Chronic Dis 32:139–144
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. Am J Human Genet 19: 133–161
Kelsey JL, Whittemore AS, Evans AS, Thompson WD (1996) Methods in observational epidemiology, 2nd edn. Oxford University Press, New York
Khoury MJ, Beaty TH (1994) Applications of the case-control method in genetic epidemiology. Epidemiol Rev 16:134–150
Kupper LL, McMichael AJ, Spirtas R (1975) A hybrid epidemiologic study design useful in estimating relative risk. J Am Stat Assoc 70:524–528
Lane-Claypon JE (1926) A further report on cancer of the breast. Her Majesty’s Stationery Office, London
Langholz B, Borgan O (1995) Counter-matching: a stratified nested case-control sampling method. Biometrika 82:69–79
Langholz B, Borgan O (1997) Estimation of absolute risk from nested case-control data. Biometrics 53:767–774
Langholz B, Goldstein L (1996) Risk set sampling in epidemiologic cohort studies. Stat Sci 11:35–53
Levin ML, Goldstein H, Gerhardt PR (1950) Cancer and tobacco smoking. A preliminary report. J Am Med Assoc 143:336–338
Liddell FDK, McDonald JC, Thomas DC (1977) Methods of cohort analysis: appraisal by application to asbestos mining. J R Stat Soc (Ser A) 140:469–491
Lilienfeld AM, Lilienfeld DE (1979) A century of case-control studies: progress? J Chronic Dis 32:5–13
Lin DY, Ying Z (1993) Cox regression with incomplete covariate measurements. J Am Stat Assoc 88:1341–1349
Little RJA, Rubin DB (2002) Statistical analysis with missing data, 2nd edn. Wiley, New York
Lombard HL, Doering CR (1928) Cancer studies in Massachusetts. 2. Habits, characteristics and environment of individuals with and without cancer. N Engl J Med 198:481–487
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. Bull World Health Org 43:209–221
Mantel N, Haenszel W (1959) Statistical aspects of the analysis of data from retrospective studies of disease. J Natl Cancer Inst 22:719–748
Miettinen OS (1970) Matching and design efficiency in retrospective studies. Am J Epidemiol 91:111–118
Miettinen O (1976) Estimability and estimation in case-referent studies. Am J Epidemiol 103: 226–235
Miettinen O (1982) Design options in epidemiologic research: an update. Scand J Work Environ Health 8:7–14
Miettinen OS (1985) Theoretical epidemiology: principles of occurrence research in medicine. Wiley, New York
Neutra RR, Drolette ME (1978) Estimating exposure-specific disease rates from case-control studies using Bayes theorem. Am J Epidemiol 108:214–222
Neyman J (1955) Statistics – servant of all sciences. Science 122:401–406
O’Neil MJ (1979) Estimating the nonresponse bias due to refusals in telephone surveys. Public Opin Q 43:218–232
Poole C (1987) Critical appraisal of the exposure-potential restriction rule. Am J Epidemiol 125:179–183
Prentice RL (1986) A case-cohort design for epidemiologic cohort studies and disease prevention trials. Biometrika 73:1–11
Prentice RL (1996) Measurement error and results from analytic epidemiology: dietary fat and breast cancer. J Natl Cancer Inst 88:1738–1747
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. Int J Cancer 16:376–383
Robins J, Pike M (1990) The validity of case-control studies with nonrandom selection of controls. Epidemiology 1:273–284
Robins JM, Gail MH, Lubin JH (1986) More on ‘biased selection of controls for case-control analyses of cohort studies’. Biometrics 42:293–29
Robison LL, Daigle A (1984) Control selection using random digit dialing for cases of childhood cancer. Am J Epidemiol 120:164–165
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. Int J Epidemiol 19:205–213
Rosenbaum PR (1987) The role of a second control group in an observational study (with discussion). Stat Sci 2:292–316
Rothman KJ (1986) Modern epidemiology. Little, Brown, Boston
Rothman KJ, Greenland S (1998) Modern epidemiology, 2nd edn. Lippincott-Raven, Philadelphia
Schlesselman JJ (1982) Case-control studies. Oxford University Press, New York
Schlesselman JJ, Stadel BV (1987) Exposure opportunity in epidemiologic studies. Am J Epidemiol 125:174–178
Sheehe PR (1962) Dynamic risk analysis in retrospective matched pair studies of disease. Biometrics 18:323–341
Smith DC, Prentice R, Thompson DJ, Herrmann W (1975) Association of exogenous estrogen and endometrial carcinoma. N Engl J Med 293:1164–1167
Smith PG, Day NE (1984) The design of case-control studies: the influence of confounding and interaction effects. Int J Epidemiol 13:356–365
Smith PG, Rodrigues LC, Fine PEM (1984) Assessment of the protective efficacy of vaccines against common diseases using case-control and cohort studies. Int J Epidemiol 13:87–93
Taubes G (1995) Epidemiology faces its limits. Science 269:164–169
Thomas DC, Greenland S (1983) The relative efficiencies of matched and independent sample designs for case-control studies. J Chronic Dis 36:685–697
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. Bull Cancer 64:45–60
Wacholder S (1995) Design issues in case-control studies. Stat Methods Med Res 4:293–309
Wacholder S, McLaughlin JK, Silverman DT, Mandel JS (1992a) Selection of controls in case-control studies I. Principles. Am J Epidemiol 135:1019–1028
Wacholder S, Silverman DT, McLaughlin JK, Mandel JS (1992b) Selection of controls in case-control studies II. Types of controls. Am J Epidemiol 135:1029–1041
Waksberg J (1978) Sampling methods for random digit dialing. J Am Stat Assoc 73:40–46
Weinberg CR, Wilcox AJ (1998) Reproductive epidemiology. In: Rothman KJ, Greenland S (eds) Modern epidemiology, 2nd edn., Chap. 29. Lippincott-Raven, Philadeplphia, pp 585–608
Weiss NS (1994) Application of the case-control method in the evaluation of screening. Epidemiol Rev 16:102–108
Weiss NS (2002) Can the ‘specificity’ of an association be rehabilitated as a basis for supporting a causal hypothesis? Epidemiology 13:6–8
White JE (1982) A two stage design for the study of the relationship between a rare exposure and a rare disease. Am J Epidemiol 115:119–128
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. J Am Med Assoc 143: 329–336
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 Res 46: 956–961
Ziel HK, Finkle WD (1975) Increased risk of endometrial carcinoma among users of conjugated estrogens. N Engl J Med 293:1167–1170
Acknowledgements
I am indebted to Sander Greenland, Noel Weiss, the editors and an anonymous referee for helpful comments on an earlier draft. This work was supported in part by grant R01-CA40644 from the US Public Health Service.
Author information
Authors and Affiliations
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2014 Springer Science+Business Media New York
About this entry
Cite this entry
Breslow, N.E. (2014). Case-Control Studies. In: Ahrens, W., Pigeot, I. (eds) Handbook of Epidemiology. Springer, New York, NY. https://doi.org/10.1007/978-0-387-09834-0_7
Download citation
DOI: https://doi.org/10.1007/978-0-387-09834-0_7
Publisher Name: Springer, New York, NY
Print ISBN: 978-0-387-09833-3
Online ISBN: 978-0-387-09834-0
eBook Packages: MedicineReference Module Medicine