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Cohort Studies Sukon Kanchanaraksa, PhD Johns Hopkins University

Design of a Cohort Study Identify: Exposed Not exposed follow: Develop Do not develop Develop Do not develop 3

Cohort Study Totals First, identify Exposed Not exposed a + b c + d 4

Cohort Study Then, follow to see whether Disease develops Disease does not develop Totals First, identify Exposed a b a + b Not exposed c d c + d 5

Cohort Study Then, follow to see whether Calculate and compare First, identify Disease develops Disease does not develop Totals Exposed a b a + b Not exposed c d c + d Incidence of a a+b c c+d a a+b = Incidence in exposed c c+d = Incidence in not exposed 6

Cohort Study Then, follow to see whether Calculate First, select 84 3000 87 5000 Smoke cigarettes Do not smoke cigarettes Develop CHD Do not develop CHD Totals 84 2916 3000 87 4913 5000 = 0.028 = Incidence in 'smoke cigarettes' Incidence of 84 3000 87 5000 = 0.0174 = Incidence in 'not smoke cigarettes' 7

Design of a Cohort Study Begin with: Defined population N o n - r a n d o m i z e d Identify : Exposed Not exposed follow: Develop Do not develop Develop Do not develop 8

Comparison of Experimental vs. Observational Study Experimental (Randomized trial) Observational (Cohort study) Population Population Random allocation Other-than-random allocation Group A Group B Group A Group B 9

Types of Cohort Studies Prospective cohort study Concurrent cohort study or longitudinal study Retrospective cohort study Non-concurrent cohort or historical cohort study = Investigator 10

Time Frames for a Hypothetical Prospective Cohort Study Conducted in 2000 Prospective 2000 Defined population N o n - r a n d o m i z e d Exposed Not exposed Disease No Disease No 11

Time Frames for a Hypothetical Retrospective Cohort Study Conducted in 2000 Defined population N o n - r a n d o m i z e d Retrospective 1980 Exposed Not exposed Disease No Disease No 2000 12

Differentiating between Prospective and Retrospective Prospective cohort study Investigator Starts the study (from the beginning) with the identification of the population and the exposure status (exposed/not exposed groups) Follows them (over time) for the development of Takes a relatively long time to complete the study (as long as the length of the study) 13

Differentiating between Prospective and Retrospective Retrospective cohort study Investigator Uses existing data collected in the past to identify the population and the exposure status (exposed/not exposed groups) Determines at present the (development) status of Investigator spends a relatively short time to: Assemble study population (and the exposed/not exposed groups) from past data Determine status at the present time (no future follow-up) 14

Combined Prospective and Retrospective Cohort Study Investigator uses existing data collected in the past to: Identify the population and the exposure status (exposed/not exposed groups) Follow them into the future for the development of the Investigator Spends a relatively short time to assemble study population (and the exposed/not exposed groups) from past data Will spend additional time following them into the future for the development of 15

Example of a Prospective Cohort Study: Framingham Study Prospective 1948 Defined Framingham study population N o n - r a n d o m i z e d Exposed Not exposed Disease No Disease No 16

Framingham Study Objectives To study the impact of several factors on incidence of cardiovascular s Exposures Blood pressure, smoking, body weight, diabetes, exercise, etc. Multiple Outcomes Coronary heart, stroke, congestive heart failure, peripheral arterial 17

Framingham Study as a Cohort Study The study started with a defined population Investigators (USPHS and NHLBI) started by identifying a new population and did not use existing data to identify the population and the exposure groups There were several hypotheses to be tested Different exposures and different outcomes For each exposure, investigators identified the exposed and the not exposed groups For each exposure, the participants were followed for the development of Different exposures were studied, as well as different s 18

Derivation of the Framingham Study Population Random sample Men Women Total 3074 3433 6507 Respondents 2024 2445 4469 Volunteers 312 428 740 Respondents free of CHD* 1975 2418 4393 Volunteers free of CHD 307 427 734 Total Free of CHD 2282 2845 5127 * CHD = coronary heart 19

Follow-Up of Participants Risk factors and the development of cardiovascular events were evaluated every two years by medical history, medical record review, and physical examination All diagnoses were verified without knowledge of risk factors by Framingham examiners who reviewed medical records and death certificates Approximately three percent of the subjects were lost to follow-up for mortality during the first 45 years of the study 20

Timeline of Milestones from the Framingham Study 1948: start of the Framingham Heart Study 1960: cigarette smoking found to increase risk of heart 1961: cholesterol, blood pressure, and ECG abnormalities found to increase risk of heart 1965: first Framingham Heart Study report on stroke 1967: physical activity found to reduce risk of heart ; obesity to increase the risk 1970: high blood pressure found to increase the risk of stroke 1974: diabetes found to be associated with cardiovascular More milestones: www.framingham.com/heart/timeline.htm http://www.framingham.com/heart/timeline.htm 21

Book A Change of Heart: How the People of Framingham, Massachusetts, Helped Unravel the Mysteries of Cardiovascular Disease 22

Average Annual Incidence of Coronary Heart Disease by Weight, Gender, and Age Group Rate per 1,000 20 10 0 30 20 Women Men 10 Above median weight 0 Below median weight 40-49 50-59 60-69 70-79 Age group 23

Average Annual Incidence of Coronary Heart Disease by Systolic Blood Pressure Rate per 1,000 30 25 20 15 10 5 0 Men Women <120 120 139 140 159 160 179 Systolic blood pressure, mm Hg 180+ 24

CHD Risk Assessment Based on Relationship Between HDL and LDL Cholesterol Men 50 70 Years Relative Risk 3 2 1 Average risk 0 HDL < 26 mg/dl HDL < 56 mg/dl HDL < 86 mg/dl 100 160 220 LDL cholesterol (mg/dl) Source: Kannel WB. CHD Risk Factors: a Framingham Study Update. Hosp Pract (Off Ed) 1990;25:119-127. 25

Types of Potential Bias in Cohort Studies Selection bias Select participants into exposed and not exposed groups based on some characteristics that may affect the outcome Information bias Collect different quality and extent of information from exposed and not exposed groups Loss to follow-up differs between exposed and not exposed (or between and no ) Misclassification bias Misclassify exposure status or status 26

When Is a Cohort Study Warranted? When the (alleged) exposure is known When exposure is rare and incidence of among exposed is high (even if the exposure is rare, determined investigators will identify exposed individuals) When the time between exposure and is relatively short When adequate funding is available When the investigator has a long life expectancy 27

Review What are the differences in the study design between prospective cohort study and retrospective cohort study? What are the differences in the study design between randomized clinical trial study and cohort study? Why is cohort study preferred for studying rare exposure? 28