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  • 1.  proper name of a retrospective matched cohort study?

    Posted 02-15-2017 02:12
    A common method used in clinical research involving retrospective data is a
    retrospective cohort study with a “control” cohort matched to a “case”
    cohort so the cohorts are “similar” on important (potentially confounding)
    variables. This is often mislabeled as a “case-control study” in the
    medical community, but it is not in the epidemiological sense. Does
    someone know an accurate name for these kinds of studies that is true to
    the epidemiology, and reflects the observational retrospective nature of
    these artificially-matched cohort studies? Even a nice catchy name that
    distinguishes it from a true case-control study would be nice.


    Raoul J. Burchette, MA, MS
    Biostatistician III
    Biostatistics, Programming & Research Database Services


    Kaiser Permanente, Regional Offices
    Department of Research and Evaluation
    100 S. Los Robles Ave., 3rd Floor (32W07)
    Pasadena, CA 91101


    (626) 564-3471 (message)
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    Raoul.J.Burchette@kp.org (email)
    ---------
    kp.org/thrive


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  • 2.  RE: proper name of a retrospective matched cohort study?

    Posted 02-16-2017 05:12
    I would call this a matched cohort study or a quasi-experiment.

    Francis C. Dane
    Chair of Arts & Sciences
    Jefferson College of Health Sciences

    Sent from my iPhone

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  • 3.  RE: proper name of a retrospective matched cohort study?

    Posted 02-16-2017 10:26

    Raoul,

    You bring up an excellent issue about how studies are named depending on how the data set was obtained.  I share your observation that “case” and “control” is often used in other than the most stringent definitions in epidemiology and medical research.  Reading your description also raises some other questions.  You may (or may not…) have a case-cohort study, see my elaboration below.

    In your case, the data including covariates and outcome information already exists and, as you point out, it is clearly a retrospective study.  It is not entirely clear to me what you mean by “controls” that are matched to “cases” that are similar on important variables, though.  All these studies are concerned about relating outcome to exposures in one way or another.  When identifying those who developed the condition (“cases”) to those who did not (“controls”) one matches (or otherwise builds two groups) based on outcome.  One can then investigate whether certain exposures (covariates) seem to be systematically different for cases as opposed to controls.  On the other hand, one can match on “important covariates” and investigate whether a certain exposure to something of specific interest seems to be associated with the different outcomes of interest in otherwise comparable individuals.  Example:  You do procedure X in some patients that you see in your hospital for a special condition and want to see whether that procedure seems beneficial with respect to some outcome Y.  Since this is observational data, it is likely that those who receive procedure X are systematically different from those who do not.  Procedure X is the exposure of interest and you can find comparable patients by matching on the exposure (procedure X), often done by identifying all those who received procedure X and finding patients who did not but are similar with respect to age, race, gender, important preexisting conditions, time since onset of symptoms, whether transferred from some other hospital, insurance status, etc.  (often propensity score matching is used but other techniques are available as well).  One can then see whether exposure X is associated with outcome Y.  Note that this is fundamentally different from matching on the outcome Y and that it does *not* control for outcome Y but for exposure X instead.

    Your description could be read as implying that matching was both, on outcome Y *and* on important covariates X and that has the potential to destroy the information you are looking for.  Consider this: You pick “cases” and “controls” and make sure that they have comparable traits like age, gender, etc.  Now: What can you learn about the relationship of gender with outcome Y? Really not much, because you manufactured the data such that traits follow a specific distribution that you decided on.

    Let’s assume that “cases” and “controls” where *not* also matched on covariates that are to be investigated in this study.  There is the approach of case-cohort study that uses all cases and combines them with a sub-cohort of controls.  A good motivation for doing so would be that covariate information is costly to obtain and therefore all controls cannot be assessed (e.g., additional lab-work using stored specimen/samples from the participants/patients).  (If all covariates are known for all controls anyhow, one would need specific reasons why one would not adjust for differences in known covariates by other means like regression or inverse probability weighting based on propensity scores instead of disregarding some participants completely – namely those who are not matched, which is regularly are very large group.)

    Reference to case-cohort studies:

    Prentice RL. A case-cohort design for epidemiologic cohort studies and disease prevention trials. Biometrika. 1986;73(1):1-11. doi: 10.1093/biomet/73.1.1.

    Kalbfleisch JD, Prentice RL. The Statistical Analysis of Failure Time Data. Second ed. Hoboken, New Jersey: Wiley; 2002.

    Reference to epidemiological study design (overview):

    Rothman KJ, Greenland S, Lash TL. Modern Epidemiology. Third ed. Philadelphia: Lippincott Williams & Wilkins; 2008. (Chapters 6, 7, 8; note especially the remarks on p. 112 regarding “controls” – for a correct interpretation of what you are estimating you will have to pay attention to whether “controls” were “disease-free” but at risk at the beginning of the study period,  whether they were “disease-free” at the point in time when the respective “case” occurred (e.g., did not have colorectal cancer by the case’s age 54, but possibly could developed the condition later as well), or whether they were required to be “disease-free” throughout the entire study period irrespective).

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    Fridtjof Thomas, Ph.D.
    Associate Professor, Division of Biostatistics
    College of Medicine/Department of Preventive Medicine
    University of Tennessee Health Science Center

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  • 4.  RE: proper name of a retrospective matched cohort study?

    Posted 02-16-2017 20:02

    Raoul, that is an excellent question you ask.

    You could call it "retrospective matched-cohort study" like you already do, or "matched historical-cohort study", or some other variant of those. Also, to call it a "retrospective-cohort study with two matched cohorts" would be wordy but accurate.

    I think I have seen it called "matched-cohort study" in one paper.

    In a paper that I and my colleagues currently have in press, we simply called it a retrospective cohort study, but then we took an additional five lines of text to explain how our study had two planned cohorts & how we had 2:1 matching of control-cohort subjects to case-cohort subjects.  In retrospect, I wish we'd come up with a better name to call it.


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  • 5.  RE: proper name of a retrospective matched cohort study?

    Posted 02-17-2017 16:19
    As I understand it, a cohort is a group of patients defined by an exposure. A case is a patient who has a particular outcome. So the phrase "case cohort" is ambiguous. If you are mining the electronic health records and select every child who received aspirin to control a fever and compare them to a control group of children who received acetaminophen to control a fever with the hypothesis that Reye's syndrome is more likely in the aspirin cohort, that's a retrospective cohort design. If you are mining the same electronic health records and select every child who came down with Reye's syndrome and compare them to a control group of children who did not experience Reye's syndrome with the hypothesis that aspirin was taken more often among the Reye's syndrome group, that's a case control design.

    It gets a little bit tricky because context is important. Low birth weight is an outcome to an obstetrician, but it is an exposure to a neonatologist. But if there is a temporal order where A precedes B in time and A is thought to be a cause of B, then defining your controls as not A makes it a cohort study and defining your controls as not B makes is a case control study.

    I suspect you are using the word "cohort" in its generic sense meaning a group of people who share something in common. Or maybe you are using the word "case" in its generic sense meaning an instance. But if you are defining a case cohort from an Epidemiologic perspective, I would argue that it represents the intersection of A and B (all children who take aspirin AND develop Reye's syndrome). If you think about this long enough, you realize that a case cohort makes no sense from a statistical perspective because you're going to end up missing one of the four cells in the two by two classification of A and B.

    Another way of thinking about it is the direction of travel. If you retrospectively select on B and not B and go further back in time to see who does or does not have A, you have a case control design. If you retrospectively select on A and not A and then move forward in time (but not all the way back to the present) to see who does or does not have B, you have a retrospective cohort design.

    Now there's such a thing as a nested case control design. So you study a cohort versus a control (usually prospectively) and you find that some of your cohort develop your outcome and some do not. You'd like to run some additional tests (e.g., genetics) on all of the cases within your cohort, but it's too expensive to run the tests on all the non-cases within your cohort. So you get a subset of non-cases from your cohort and because your sample size is smaller, you do some matching. So you have a case control design nested within a cohort design. Your hypothesis is that a positive result on the expensive test is more likely among cases than controls. Note that both cases and controls are drawn from the exposed cohort--the unexposed control cohort patients do not participate in a nested case-control study.

    It's also possible to nest a case control design within a randomized trial. Select all of the patients randomized to the active arm AND who experience the outcome of interest. Run a super expensive test on these patients. Now pick a small but carefully matched set of patients in the active arm who did not experience the outcome of interest and run those same tests.

    To make things even more complicated, there is a case-cohort design and a case-crossover design. See 7.2 - Advanced Case-Control Designs | STAT 507 for details. I think I'll stop here before my head explodes.

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    Stephen Simon, blog.pmean.com
    Independent Statistical Consultant
    P. Mean Consulting
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