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Causal inference and case-control studies with applications related to childhood diabetes
Umeå University, Faculty of Social Sciences, Umeå School of Business and Economics (USBE), Statistics.
2014 (English)Doctoral thesis, comprehensive summary (Other academic)Alternative title
Kausal inferens och fall-kontroll studier med applikationer inom barndiabetes (Swedish)
Abstract [en]

This thesis contributes to the research area of causal inference, where estimation of the effect of a treatment on an outcome of interest is the main objective. Some aspects of the estimation of average causal effects in observational studies in general, and case-control studies in particular, are explored.

An important part of estimating causal effects in an observational study is to control for covariates. The first paper of this thesis concerns the selection of minimal covariate sets sufficient for unconfoundedness of the treatment assignment. A data-driven implementation of two covariate selection algorithms is proposed and evaluated.

A common sampling scheme in epidemiology, and when investigating rare events, is the case-control design. In the second paper we study estimators of the marginal causal odds ratio in matched and independent case-control designs. Estimators that, under a logistic regression model, utilize information about the known prevalence of being a case is examined and compared through simulations.

The third paper investigates the particular situation where case-control sampled data is reused to estimate the effect of the case-defining event on an outcome of interest. The consequence of ignoring the design when estimating the average causal effect is discussed and a design-weighted matching estimator is proposed. The performance of the estimator is evaluated with simulation experiments, when matching on the covariates directly and when matching on the propensity score.

The last paper studies the effect of type 1 diabetes mellitus (T1DM) on school achievements using data from the Swedish Childhood Diabetes Register, a population-based incidence register. We apply theoretical results from the second and third papers in the estimation of the average causal effect within the T1DM population. A matching estimator that accounts for the matched case-control design is used.

Place, publisher, year, edition, pages
Umeå: Umeå universitet , 2014. , p. 22
Series
Statistical studies, ISSN 1100-8989 ; 48
Keywords [en]
covariate selection, design-weighted estimation, marginal effect, matching, register study, treatment effect, type 1 diabetes mellitus
National Category
Probability Theory and Statistics
Research subject
Statistics
Identifiers
URN: urn:nbn:se:umu:diva-94993ISBN: 978-91-7601-151-5 (print)OAI: oai:DiVA.org:umu-94993DiVA, id: diva2:757008
Public defence
2014-11-21, Hörsal F, Humanisthuset, Umeå universitet, Umeå, 10:15 (English)
Opponent
Supervisors
Available from: 2014-10-24 Created: 2014-10-20 Last updated: 2018-06-07Bibliographically approved
List of papers
1. Data-driven algorithms for dimension reduction in causal inference
Open this publication in new window or tab >>Data-driven algorithms for dimension reduction in causal inference
2017 (English)In: Computational Statistics & Data Analysis, ISSN 0167-9473, E-ISSN 1872-7352, Vol. 105, p. 280-292Article in journal (Refereed) Published
Abstract [en]

In observational studies, the causal effect of a treatment may be confounded with variables that are related to both the treatment and the outcome of interest. In order to identify a causal effect, such studies often rely on the unconfoundedness assumption, i.e., that all confounding variables are observed. The choice of covariates to control for, which is primarily based on subject matter knowledge, may result in a large covariate vector in the attempt to ensure that unconfoundedness holds. However, including redundant covariates can affect bias and efficiency of nonparametric causal effect estimators, e.g., due to the curse of dimensionality. In this paper, data-driven algo- rithms for the selection of sufficient covariate subsets are investigated. Under the assumption of unconfoundedness we search for minimal subsets of the covariate vector. Based on the framework of sufficient dimension reduction or kernel smoothing, the algorithms perform a backward elim- ination procedure testing the significance of each covariate. Their performance is evaluated in simulations and an application using data from the Swedish Childhood Diabetes Register is also presented.

Keywords
covariate selection, marginal co-ordinate hypothesis test, matching, kernel smoothing, type 1 diabetes mellitus
National Category
Probability Theory and Statistics
Research subject
Statistics
Identifiers
urn:nbn:se:umu:diva-80696 (URN)10.1016/j.csda.2016.08.012 (DOI)000385604500019 ()2-s2.0-84987932350 (Scopus ID)
Funder
Swedish National Infrastructure for Computing (SNIC), SNIC 2016/1-2Swedish Research Council, 2013-672Swedish Research Council, 07531Riksbankens Jubileumsfond, P11-0814:1
Available from: 2013-09-24 Created: 2013-09-24 Last updated: 2023-03-23Bibliographically approved
2. Estimating a marginal causal odds ratio in a case-control design: analyzing the effect of low birth weight on the risk of type 1 diabetes mellitus
Open this publication in new window or tab >>Estimating a marginal causal odds ratio in a case-control design: analyzing the effect of low birth weight on the risk of type 1 diabetes mellitus
2013 (English)In: Statistics in Medicine, ISSN 0277-6715, E-ISSN 1097-0258, Vol. 32, no 14, p. 2500-2512Article in journal (Refereed) Published
Abstract [en]

Estimation of marginal causal effects from case-control data has two complications: (i) confounding due to the fact that the exposure under study is not randomized, and (ii) bias from the case-control sampling scheme. In this paper, we study estimators of the marginal causal odds ratio, addressing these issues for matched and unmatched case-control designs when utilizing the knowledge of the known prevalence of being a case. The estimators are implemented in simulations where their finite sample properties are studied and approximations of their variances are derived with the delta method. Also, we illustrate the methods by analyzing the effect of low birth weight on the risk of type 1 diabetes mellitus using data from the Swedish Childhood Diabetes Register, a nationwide population-based incidence register.

Keywords
causal effect, incidence register, potential outcomes, design weighting
National Category
Probability Theory and Statistics
Research subject
Statistics
Identifiers
urn:nbn:se:umu:diva-68956 (URN)10.1002/sim.5826 (DOI)000319880100013 ()2-s2.0-84878649398 (Scopus ID)
Funder
Riksbankens Jubileumsfond, P11-0814:1Swedish Research Council, 0735
Available from: 2013-05-02 Created: 2013-05-02 Last updated: 2023-03-23Bibliographically approved
3. Estimating marginal causal effects in a secondary analysis of case-control data
Open this publication in new window or tab >>Estimating marginal causal effects in a secondary analysis of case-control data
2017 (English)In: Statistics in Medicine, ISSN 0277-6715, E-ISSN 1097-0258, Vol. 36, no 15, p. 2404-2419Article in journal (Refereed) Published
Abstract [en]

When an initial case-control study is performed, data can be used in a secondary analysis to evaluate the effect of the case-defining event on later outcomes. In this paper, we study the example in which the role of the event is changed from a response variable to a treatment of interest. If the aim is to estimate marginal effects, such as average effects in the population, the sampling scheme needs to be adjusted for. We study estimators of the average effect of the treatment in a secondary analysis of matched and unmatched case-control data where the probability of being a case is known. For a general class of estimators, we show the components of the bias resulting from ignoring the sampling scheme and demonstrate a design-weighted matching estimator of the average causal effect. In simulations, the finite sample properties of the design-weighted matching estimator are studied. Using a Swedish diabetes incidence register with a matched case-control design, we study the effect of childhood onset diabetes on the use of antidepressant medication as an adult.

Place, publisher, year, edition, pages
Hoboken: Wiley-Blackwell, 2017
Keywords
design-weighted estimation, matched case-control study, propensity score
National Category
Probability Theory and Statistics Public Health, Global Health and Social Medicine
Research subject
Statistics
Identifiers
urn:nbn:se:umu:diva-94965 (URN)10.1002/sim.7277 (DOI)000402799900007 ()28276084 (PubMedID)2-s2.0-85014944257 (Scopus ID)
Funder
Swedish Research Council, 07531Riksbankens Jubileumsfond, P11-0814:1
Available from: 2014-10-20 Created: 2014-10-20 Last updated: 2025-02-20Bibliographically approved
4. Effect of type 1 diabetes on school performance in a dynamic world: new analysis exploring Swedish register data
Open this publication in new window or tab >>Effect of type 1 diabetes on school performance in a dynamic world: new analysis exploring Swedish register data
2019 (English)In: Applied Economics, ISSN 0003-6846, E-ISSN 1466-4283, Vol. 51, no 24, p. 2606-2622Article in journal (Refereed) Published
Abstract [en]

This paper investigates if the effect of type 1 diabetes mellitus (T1DM) on school performance, documented in prior research, has changed in more recent birth cohorts of children using national Swedish population register data. The issue is of interest because management and treatment of the disease have improved over the last decades and, furthermore, because of changes in the educational grading system. Despite these changes, data indicate a persistent negative effect of T1DM on compulsory and upper secondary school grades with a standardized effect size of −0.109 and −0.070, respectively, and the results appear only marginally smaller compared to earlier findings in cohorts completing school under the previous grading system. Moreover, the results are consistent for alternative model specifications and econometric estimation strategies. Whereas access to new treatment technologies and improved diabetes management strategies has reduced the burden of diabetes in daily life, the results from this study indicate that continued efforts are needed to improve the situation in school for children with T1DM to prevent potential long-term socio-economic consequences.

Place, publisher, year, edition, pages
Taylor & Francis Group, 2019
Keywords
Type 1 diabetes, education, school performance, register data
National Category
Peace and Conflict Studies Other Social Sciences not elsewhere specified Economics
Identifiers
urn:nbn:se:umu:diva-94969 (URN)10.1080/00036846.2018.1558347 (DOI)000459830200003 ()2-s2.0-85059071387 (Scopus ID)
Note

Originally included in thesis in manuscript form.

Available from: 2014-10-20 Created: 2014-10-20 Last updated: 2025-02-20Bibliographically approved

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