474 results on '"Schomaker, Michael"'
Search Results
2. Causal evidence in health decision making: methodological approaches of causal inference and health decision science
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Kühne, Felicitas, Schomaker, Michael, Stojkov, Igor, Jahn, Beate, Conrads-Frank, Annette, Siebert, Silke, Sroczynski, Gaby, Puntscher, Sibylle, Schmid, Daniela, Schnell-Inderst, Petra, and Siebert, Uwe
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causal inference ,health decision science ,epidemiology ,decision-analytic modeling ,medical decision making ,health technology assessment ,Medicine - Abstract
Objectives: Public health decision making is a complex process based on thorough and comprehensive health technology assessments involving the comparison of different strategies, values and tradeoffs under uncertainty. This process must be based on best available evidence and plausible assumptions. Causal inference and health decision science are two methodological approaches providing information to help guide decision making in health care. Both approaches are quantitative methods that use statistical and modeling techniques and simplifying assumptions to mimic the complexity of the real world. We intend to review and lay out both disciplines with their aims, strengths and limitations based on a combination of textbook knowledge and expert experience.Methods: To help understanding and differentiating the methodological approaches of causal inference and health decision science, we reviewed both methods with the focus on aims, research questions, methods, assumptions, limitations and challenges, and software. For each methodological approach, we established a group of four experts from our own working group to carefully review and summarize each method, followed by structured discussion rounds and written reviews, in which the experts from all disciplines including HTA and medicine were involved. The entire expert group discussed objectives, strengths and limitations of both methodological areas, and potential synergies. Finally, we derived recommendations for further research and provide a brief outlook on future trends.Results: Causal inference methods aim for drawing causal conclusions from empirical data on the relationship of pre-specified interventions on a specific target outcome and apply a counterfactual framework and statistical techniques to derive causal effects of exposures or interventions from these data. Causal inference is based on a causal diagram, more specifically, a directed acyclic graph (DAG), which encodes the assumptions regarding the causal relations between variables. Depending on the type of confounding and selection bias, traditional statistical methods or more complex g-methods are needed to derive valid causal effects. Besides the correct specification of the DAG and the statistical model, assumptions such as consistency, positivity, and exchangeability must be checked when aiming at causal inference.Health decision science aims for guiding policy decision making regarding health interventions considering and balancing multiple competing objectives of a decision based on data from multiple sources and studies, for example prevalence studies, clinical trials and long-term observational routine effectiveness studies, and studies on preferences and costs. It involves decision analysis, a systematic, explicit and quantitative framework to guide decisions under uncertainty. Decision analyses are based on decision-analytic models to mimic the course of disease as well as aspects and consequences of the intervention in order to quantitatively optimize the decision. Depending on the type of decision problem, decision trees, state-transition models, discrete event simulation models, dynamic transmission models, or other model types are applied. Models must be validated against observed data, and comprehensive sensitivity analyses must be performed to assess uncertainty. Besides the appropriate choice of the model type and the valid specification of the model structure, it must be checked if input parameters of effects can be interpreted as causal parameters in the model. Otherwise results will be biased.Conclusions: Both causal inference and health decision science aim for providing best causal evidence for informed health decision making. The strengths and limitations of both methods differ and a good understanding of both methods is essential for correct application but also for correct interpretation of findings from the described methods. Importantly, decision-analytic modeling should be combined with causal inference when developing guidance and recommendations regarding decisions on health care interventions.
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- 2022
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3. Recoverability of Causal Effects in a Longitudinal Study under Presence of Missing Data
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Holovchak, Anastasiia, McIlleron, Helen, Denti, Paolo, and Schomaker, Michael
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Statistics - Methodology - Abstract
Missing data in multiple variables is a common issue. We investigate the applicability of the framework of graphical models for handling missing data to a complex longitudinal pharmacological study of children with HIV treated with an efavirenz-based regimen as part of the CHAPAS-3 trial. Specifically, we examine whether the causal effects of interest, defined through static interventions on multiple continuous variables, can be recovered (estimated consistently) from the available data only. So far, no general algorithms are available to decide on recoverability, and decisions have to be made on a case-by-case basis. We emphasize sensitivity of recoverability to even the smallest changes in the graph structure, and present recoverability results for three plausible missingness directed acyclic graphs (m-DAGs) in the CHAPAS-3 study, informed by clinical knowledge. Furthermore, we propose the concept of "closed missingness mechanisms" and show that under these mechanisms an available case analysis is admissible for consistent estimation for any type of statistical and causal query, even if the underlying missingness mechanism is of missing not at random (MNAR) type. Both simulations and theoretical considerations demonstrate how, in the assumed MNAR setting of our study, a complete or available case analysis can be superior to multiple imputation, and estimation results vary depending on the assumed missingness DAG. Our analyses are possibly the first to show the applicability of missingness DAGs (m-DAGs) to complex longitudinal real-world data, while highlighting the sensitivity with respect to the assumed causal model., Comment: Corrected minor typos; added Monte Carlo confidence intervals
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- 2024
4. Causal Fair Machine Learning via Rank-Preserving Interventional Distributions
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Bothmann, Ludwig, Dandl, Susanne, and Schomaker, Michael
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Computer Science - Machine Learning ,Computer Science - Computers and Society ,Statistics - Machine Learning - Abstract
A decision can be defined as fair if equal individuals are treated equally and unequals unequally. Adopting this definition, the task of designing machine learning (ML) models that mitigate unfairness in automated decision-making systems must include causal thinking when introducing protected attributes: Following a recent proposal, we define individuals as being normatively equal if they are equal in a fictitious, normatively desired (FiND) world, where the protected attributes have no (direct or indirect) causal effect on the target. We propose rank-preserving interventional distributions to define a specific FiND world in which this holds and a warping method for estimation. Evaluation criteria for both the method and the resulting ML model are presented and validated through simulations. Experiments on empirical data showcase the practical application of our method and compare results with "fairadapt" (Ple\v{c}ko and Meinshausen, 2020), a different approach for mitigating unfairness by causally preprocessing data that uses quantile regression forests. With this, we show that our warping approach effectively identifies the most discriminated individuals and mitigates unfairness.
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- 2023
5. Causal Inference for Continuous Multiple Time Point Interventions
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Schomaker, Michael, McIlleron, Helen, Denti, Paolo, and Díaz, Iván
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Statistics - Methodology ,Statistics - Applications - Abstract
There are limited options to estimate the treatment effects of variables which are continuous and measured at multiple time points, particularly if the true dose-response curve should be estimated as closely as possible. However, these situations may be of relevance: in pharmacology, one may be interested in how outcomes of people living with -- and treated for -- HIV, such as viral failure, would vary for time-varying interventions such as different drug concentration trajectories. A challenge for doing causal inference with continuous interventions is that the positivity assumption is typically violated. To address positivity violations, we develop projection functions, which reweigh and redefine the estimand of interest based on functions of the conditional support for the respective interventions. With these functions, we obtain the desired dose-response curve in areas of enough support, and otherwise a meaningful estimand that does not require the positivity assumption. We develop $g$-computation type plug-in estimators for this case. Those are contrasted with g-computation estimators which are applied to continuous interventions without specifically addressing positivity violations, which we propose to be presented with diagnostics. The ideas are illustrated with longitudinal data from HIV positive children treated with an efavirenz-based regimen as part of the CHAPAS-3 trial, which enrolled children $<13$ years in Zambia/Uganda. Simulations show in which situations a standard g-computation approach is appropriate, and in which it leads to bias and how the proposed weighted estimation approach then recovers the alternative estimand of interest.
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- 2023
6. The Delta-Method and Influence Function in Medical Statistics: a Reproducible Tutorial
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Zepeda-Tello, Rodrigo, Schomaker, Michael, Maringe, Camille, Smith, Matthew J., Belot, Aurelien, Rachet, Bernard, Schnitzer, Mireille E., and Luque-Fernandez, Miguel Angel
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Statistics - Methodology - Abstract
Approximate statistical inference via determination of the asymptotic distribution of a statistic is routinely used for inference in applied medical statistics (e.g. to estimate the standard error of the marginal or conditional risk ratio). One method for variance estimation is the classical Delta-method but there is a knowledge gap as this method is not routinely included in training for applied medical statistics and its uses are not widely understood. Given that a smooth function of an asymptotically normal estimator is also asymptotically normally distributed, the Delta-method allows approximating the large-sample variance of a function of an estimator with known large-sample properties. In a more general setting, it is a technique for approximating the variance of a functional (i.e., an estimand) that takes a function as an input and applies another function to it (e.g. the expectation function). Specifically, we may approximate the variance of the function using the functional Delta-method based on the influence function (IF). The IF explores how a functional $\phi(\theta)$ changes in response to small perturbations in the sample distribution of the estimator and allows computing the empirical standard error of the distribution of the functional. The ongoing development of new methods and techniques may pose a challenge for applied statisticians who are interested in mastering the application of these methods. In this tutorial, we review the use of the classical and functional Delta-method and their links to the IF from a practical perspective. We illustrate the methods using a cancer epidemiology example and we provide reproducible and commented code in R and Python using symbolic programming. The code can be accessed at https://github.com/migariane/DeltaMethodInfluenceFunction
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- 2022
7. Implementation of a comprehensive clinical risk management system in a university hospital
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Buchberger, Wolfgang, Schmied, Marten, Schomaker, Michael, del Rio, Anca, and Siebert, Uwe
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- 2024
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8. Regression and Causality
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Schomaker, Michael
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Statistics - Methodology ,Mathematics - Statistics Theory - Abstract
The causal effect of an intervention (treatment/exposure) on an outcome can be estimated by: i) specifying knowledge about the data-generating process; ii) assessing under what assumptions a target quantity, such as for example a causal odds ratio, can be identified given the specified knowledge (and given the measured data); and then, iii) using appropriate statistical estimation techniques to estimate the desired parameter of interest. As regression is the cornerstone of statistical analysis, it seems obvious to ask: is it appropriate to use estimated regression parameters for causal effect estimation? It turns out that using regression for effect estimation is possible, but typically requires more assumptions than competing methods. This manuscript provides a comprehensive summary of the assumptions needed to identify and estimate a causal parameter using regression and, equally important, discusses the resulting implications for statistical practice.
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- 2020
9. Estimating the Effect of Central Bank Independence on Inflation Using Longitudinal Targeted Maximum Likelihood Estimation
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Baumann, Philipp F. M., Schomaker, Michael, and Rossi, Enzo
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Economics - Econometrics ,Statistics - Applications - Abstract
The notion that an independent central bank reduces a country's inflation is a controversial hypothesis. To date, it has not been possible to satisfactorily answer this question because the complex macroeconomic structure that gives rise to the data has not been adequately incorporated into statistical analyses. We develop a causal model that summarizes the economic process of inflation. Based on this causal model and recent data, we discuss and identify the assumptions under which the effect of central bank independence on inflation can be identified and estimated. Given these and alternative assumptions, we estimate this effect using modern doubly robust effect estimators, i.e., longitudinal targeted maximum likelihood estimators. The estimation procedure incorporates machine learning algorithms and is tailored to address the challenges associated with complex longitudinal macroeconomic data. We do not find strong support for the hypothesis that having an independent central bank for a long period of time necessarily lowers inflation. Simulation studies evaluate the sensitivity of the proposed methods in complex settings when certain assumptions are violated and highlight the importance of working with appropriate learning algorithms for estimation.
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- 2020
10. Inference
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2022
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11. Causality
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2022
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12. Measures of Central Tendency and Dispersion
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2022
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13. Simple Random Sampling and Bootstrapping
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2022
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14. Hypothesis Testing
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2022
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15. Linear Regression
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2022
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16. Probability Distributions
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2022
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17. Random Variables
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2022
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18. Elements of Probability Theory
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2022
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19. Frequency Measures and Graphical Representation of Data
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2022
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20. Association of Two Variables
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2022
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21. Introduction and Framework
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2022
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22. Life years lost associated with mental illness: A cohort study of beneficiaries of a South African medical insurance scheme
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Ruffieux, Yann, Wettstein, Anja, Maartens, Gary, Folb, Naomi, Mesa-Vieira, Cristina, Didden, Christiane, Tlali, Mpho, Williams, Chanwyn, Cornell, Morna, Schomaker, Michael, Johnson, Leigh F., Joska, John A., Egger, Matthias, and Haas, Andreas D.
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- 2023
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23. Determinants of low health-related quality of life in patients with myelodysplastic syndromes: EUMDS Registry study
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Stojkov, Igor, Conrads-Frank, Annette, Rochau, Ursula, Arvandi, Marjan, Koinig, Karin A., Schomaker, Michael, Mittelman, Moshe, Fenaux, Pierre, Bowen, David, Sanz, Guillermo F., Malcovati, Luca, Langemeijer, Saskia, Germing, Ulrich, Madry, Krzysztof, Guerci-Bresler, Agnès, Culligan, Dominic J., Kotsianidis, Ioannis, Sanhes, Laurence, Mills, Juliet, Puntscher, Sibylle, Schmid, Daniela, van Marrewijk, Corine, Smith, Alexandra, Efficace, Fabio, de Witte, Theo, Stauder, Reinhard, and Siebert, Uwe
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- 2023
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24. Educational Note: Paradoxical Collider Effect in the Analysis of Non-Communicable Disease Epidemiological Data: a reproducible illustration and web application
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Luque-Fernandez, Miguel Angel, Schomaker, Michael, Redondo-Sanchez, Daniel, Perez, Maria Jose Sanchez, Vaidya, Anand, and Schnitzer, Mireille E.
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Statistics - Methodology - Abstract
Classical epidemiology has focused on the control of confounding but it is only recently that epidemiologists have started to focus on the bias produced by colliders. A collider for a certain pair of variables (e.g., an outcome Y and an exposure A) is a third variable (C) that is caused by both. In a directed acyclic graph (DAG), a collider is the variable in the middle of an inverted fork (i.e., the variable C in A -> C <- Y). Controlling for, or conditioning an analysis on a collider (i.e., through stratification or regression) can introduce a spurious association between its causes. This potentially explains many paradoxical findings in the medical literature, where established risk factors for a particular outcome appear protective. We use an example from non-communicable disease epidemiology to contextualize and explain the effect of conditioning on a collider. We generate a dataset with 1,000 observations and run Monte-Carlo simulations to estimate the effect of 24-hour dietary sodium intake on systolic blood pressure, controlling for age, which acts as a confounder, and 24-hour urinary protein excretion, which acts as a collider. We illustrate how adding a collider to a regression model introduces bias. Thus, to prevent paradoxical associations, epidemiologists estimating causal effects should be wary of conditioning on colliders. We provide R-code in easy-to-read boxes throughout the manuscript and a GitHub repository (https://github.com/migariane/ColliderApp) for the reader to reproduce our example. We also provide an educational web application allowing real-time interaction to visualize the paradoxical effect of conditioning on a collider http://watzilei.com/shiny/collider/.
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- 2018
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25. Using Longitudinal Targeted Maximum Likelihood Estimation in Complex Settings with Dynamic Interventions
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Schomaker, Michael, Luque-Fernandez, Miguel Angel, Leroy, Valeriane, and Davies, Mary-Ann
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Statistics - Methodology - Abstract
Longitudinal targeted maximum likelihood estimation (LTMLE) has very rarely been used to estimate dynamic treatment effects in the context of time-dependent confounding affected by prior treatment when faced with long follow-up times, multiple time-varying confounders, and complex associational relationships simultaneously. Reasons for this include the potential computational burden, technical challenges, restricted modeling options for long follow-up times, and limited practical guidance in the literature. However, LTMLE has desirable asymptotic properties, i.e. it is doubly robust, and can yield valid inference when used in conjunction with machine learning. We use a topical and sophisticated question from HIV treatment research to show that LTMLE can be used successfully in complex realistic settings and compare results to competing estimators. Our example illustrates the following practical challenges common to many epidemiological studies 1) long follow-up time (30 months), 2) gradually declining sample size 3) limited support for some intervention rules of interest 4) a high-dimensional set of potential adjustment variables, increasing both the need and the challenge of integrating appropriate machine learning methods 5) consideration of collider bias. Our analyses, as well as simulations, shed new light on the application of LTMLE in complex and realistic settings: we show that (i) LTMLE can yield stable and good estimates, even when confronted with small samples and limited modeling options; (ii) machine learning utilized with a small set of simple learners (if more complex ones can't be fitted) can outperform a single, complex model, which is tailored to incorporate prior clinical knowledge; (iii) performance can vary considerably depending on interventions and their support in the data, and therefore critical quality checks should accompany every LTMLE analysis.
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- 2018
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26. When and when not to use optimal model averaging
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Schomaker, Michael and Heumann, Christian
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Statistics - Methodology - Abstract
Traditionally model averaging has been viewed as an alternative to model selection with the ultimate goal to incorporate the uncertainty associated with the model selection process in standard errors and confidence intervals by using a weighted combination of candidate models. In recent years, a new class of model averaging estimators has emerged in the literature, suggesting to combine models such that the squared risk, or other risk functions, are minimized. We argue that, contrary to popular belief, these estimators do not necessarily address the challenges induced by model selection uncertainty, but should be regarded as attractive complements for the machine learning and forecasting literature, as well as tools to identify causal parameters. We illustrate our point by means of several targeted simulation studies.
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- 2018
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27. Virologic non-suppression and early loss to follow up among pregnant and non-pregnant adolescents aged 15-19 years initiating antiretroviral therapy in South Africa: a retrospective cohort study
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Nyakato, Patience, Schomaker, Michael, Fatti, Geoffrey, Tanser, Frank, Euvrard, Jonathan, Sipambo, Nosisa, Fox, Matthew P., Haas, Andreas D., Yiannoutsos, Constantin T., Davies, Mary-Ann, and Cornell, Morna
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Pregnant girls -- Drug therapy -- Statistics ,Highly active antiretroviral therapy -- Patient outcomes -- Statistics ,HIV infection -- Drug therapy -- Patient outcomes -- Statistics ,Health - Abstract
Introduction: Older adolescents aged 15-19 years continue to have high rates of loss to follow up (LTFU), and high rates of virologic non-suppression (VNS) compared to younger adolescents and adults. Adolescent females are at risk of pregnancy, which puts those living with HIV at a dualvulnerability. Our study assessed the factors associated with VNS and LTFU in older adolescents (including pregnant females) who initiated antiretroviral therapy (ART) in South Africa. Methods: We included adolescents aged 15-19 years initiating ART between 2004 and 2019, with [greater than or equal to] one viral load (VL) measurement between 4 and 24.5 months, and [greater than or equal to] 6 months follow-up, from six South African cohorts of the International epidemiology Databases to Evaluate AIDS-Southern Africa (IeDEA-SA). We defined VNS as VL [greater than or equal to]400 copies/ml and LTFU as not being in care for [greater than or equal to]180 days from ART start and not known as transferred out of the clinic or dead in the first 24 months on ART We examined factors associated with VNS and LTFU using Fine&Gray competing risk models. Results: We included a totalof 2733 adolescents, 415 (15.2%) males, median (IQR) age at ART start of 18.6 (17.3, 19.4) years. Among females, 585/2318 (25.2%) were pregnant. Over the 24-month follow-up, 424 (15.5%) of alladolescents experienced VNS: range (11.1% pregnant females and 20.5% males). Over half of all adolescents were LTFU before any other event could occur. The hazard of VNS reduced with increasing age and CD4 count above 200 cells/[micro]l at ART initiation among all adolescents having adjusted for allmeasured patient characteristics [adjusted sub-distribution hazard ratio (aSHR) 19 vs. 15 years: 0.50 (95% CI: 0.36, 0.68), aSHR: >500 vs. =200 cells/[micro]l: 0.22 (95% CI: 0.16, 0.31)]. The effect of CD4 count persisted in pregnant females. Increasing age and CD4 count >200 cells/[micro]l were risk factors for LTFU among alladolescents. Conclusions: Older adolescents had a high risk of LTFU shortly after ART start and a low risk of VNS, especially those initiating treatment during pregnancy. Interventions addressing adherence and retention should be incorporated into adolescent-friendly services to prevent VNS and LTFU and endeavour to trace lost adolescents as soon as they are identified. Keywords: adolescents; antiretroviraltherapy; HIV; loss to follow up; pregnancy; virologic non-suppression, 1 | INTRODUCTION In 2019, about 1.7 (1.1-2.4) million adolescents aged 10-19 years and 3.4 million youth aged 15-24 years were living with HIV worldwide [1], with the majority living [...]
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- 2022
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28. Correcting mortality estimates among children and youth on antiretroviral therapy in southern Africa: A comparative analysis between a multi‐country tracing study and linkage to a health information exchange.
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Nyakato, Patience, Schomaker, Michael, Boulle, Andrew, Euvrard, Jonathan, Wood, Robin, Eley, Brian, Prozesky, Hans, Christ, Benedikt, Anderegg, Nanina, Ayakaka, Irene, Rafael, Idiovino, Kunzekwenyika, Cordelia, Moore, Carolyn B., van Lettow, Monique, Chimbetete, Cleophas, Mbewe, Safari, Ballif, Marie, Egger, Matthias, Yiannoutsos, Constantin T., and Cornell, Morna
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Objectives: The objective of this study is to assess the outcomes of children, adolescents and young adults with HIV reported as lost to follow‐up, correct mortality estimates for children, adolescents and young adults with HIV for unascertained outcomes in those loss to follow‐up (LTFU) based on tracing and linkage data separately using data from the International epidemiology Databases to Evaluate AIDS in Southern Africa. Methods: We included data from two different populations of children, adolescents and young adults with HIV; (1) clinical data from children, adolescents and young adults with HIV aged ≤24 years from Lesotho, Malawi, Mozambique, Zambia and Zimbabwe; (2) clinical data from children, adolescents and young adults with HIV aged ≤14 years from the Western Cape (WC) in South Africa. Outcomes of patients lost to follow‐up were available from (1) a tracing study and (2) linkage to a health information exchange. For both populations, we compared six methods for correcting mortality estimates for all children, adolescents and young adults with HIV. Results: We found substantial variations of mortality estimates among children, adolescents and young adults with HIV reported as lost to follow‐up versus those retained in care. Ascertained mortality was higher among lost and traceable children, adolescents and young adults with HIV and lower among lost and linkable than those retained in care (mortality: 13.4% [traced] vs. 12.6% [retained‐other Southern Africa countries]; 3.4% [linked] vs. 9.4% [retained‐WC]). A high proportion of lost to follow‐up children, adolescents and young adults with HIV had self‐transferred (21.0% and 47.0%) in the traced and linked samples, respectively. The uncorrected method of non‐informative censoring yielded the lowest mortality estimates among all methods for both tracing (6.0%) and linkage (4.0%) approaches at 2 years from ART start. Among corrected methods using ascertained data, multiple imputation, incorporating ascertained data (MI(asc.)) and inverse probability weighting with logistic weights were most robust for the tracing approach. In contrast, for the linkage approach, MI(asc.) was the most robust. Conclusions: Our findings emphasise that lost to follow‐up is non‐ignorable and both tracing and linkage improved outcome ascertainment: tracing identified substantial mortality in those reported as lost to follow‐up, whereas linkage did not identify out‐of‐facility deaths, but showed that a large proportion of those reported as lost to follow‐up were self‐transfers. [ABSTRACT FROM AUTHOR]
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- 2024
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29. Comparison of Kaposi Sarcoma Risk in Human Immunodeficiency Virus-Positive Adults Across 5 Continents: A Multiregional Multicohort Study
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Judd, Ali, Zangerle, Robert, Touloumi, Giota, Warszawski, Josiane, Meyer, Laurence, Dabis, François, Krause, Murielle Mary, Ghosn, Jade, Leport, Catherine, Wittkop, Linda, Reiss, Peter, Wit, Ferdinand, Prins, Maria, Bucher, Heiner, Gibb, Diana, Fätkenheuer, Gerd, Julia, Del Amo, Obel, Niels, Thorne, Claire, Mocroft, Amanda, Kirk, Ole, Stephan, Christoph, Pérez-Hoyos, Santiago, Hamouda, Osamah, Bartmeyer, Barbara, Chkhartishvili, Nikoloz, Noguera-Julian, Antoni, Antinori, Andrea, Monforte, Antonella d’Arminio, Brockmeyer, Norbert, Prieto, Luis, Conejo, Pablo Rojo, Soriano-Arandes, Antoni, Battegay, Manuel, Kouyos, Roger, Mussini, Cristina, Tookey, Pat, Casabona, Jordi, Miró, Jose M, Castagna, Antonella, Konopnick, Deborah, Goetghebuer, Tessa, Sönnerborg, Anders, Quiros-Roldan, Eugenia, Sabin, Caroline, Teira, Ramon, Garrido, Myriam, Haerry, David, de Wit, Stéphane, Costagliola, Dominique, d’Arminio-Monforte, Antonella, del Amo, Julia, Raben, Dorthe, Chêne, Geneviève, Rojo, Conejo Pablo, Barger, Diana, Schwimmer, Christine, Termote, Monique, Campbell, Maria, Frederiksen, Casper M, Friis-Møller, Nina, Kjaer, Jesper, Brandt, Rikke Salbøl, Berenguer, Juan, Bohlius, Julia, Bouteloup, Vincent, Cozzi-Lepri, Alessandro, Davies, Mary-Anne, Dorrucci, Maria, Dunn, David, Egger, Matthias, Furrer, Hansjakob, Grabar, Sophie, Guiguet, Marguerite, Lambotte, Olivier, Leroy, Valériane, Lodi, Sara, Matheron, Sophie, Miro, Jose M, Monge, Susana, Nakagawa, Fumiyo, Paredes, Roger, Phillips, Andrew, Puoti, Massimo, Rohner, Eliane, and Schomaker, Michael
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Biomedical and Clinical Sciences ,Clinical Sciences ,Sexually Transmitted Infections ,Rare Diseases ,Emerging Infectious Diseases ,Infectious Diseases ,HIV/AIDS ,Infection ,Adolescent ,Adult ,Anti-Retroviral Agents ,CD4 Lymphocyte Count ,Cohort Studies ,Female ,HIV Infections ,HIV-1 ,Humans ,Male ,Middle Aged ,Risk Factors ,Sarcoma ,Kaposi ,Viral Load ,Young Adult ,AIDS-defining Cancer Project Working Group for IeDEA and COHERE in EuroCoord ,HIV ,Kaposi sarcoma ,antiretroviral therapy ,cohort study ,Biological Sciences ,Medical and Health Sciences ,Microbiology ,Clinical sciences - Abstract
BackgroundWe compared Kaposi sarcoma (KS) risk in adults who started antiretroviral therapy (ART) across the Asia-Pacific, South Africa, Europe, Latin, and North America.MethodsWe included cohort data of human immunodeficiency virus (HIV)-positive adults who started ART after 1995 within the framework of 2 large collaborations of observational HIV cohorts. We present incidence rates and adjusted hazard ratios (aHRs).ResultsWe included 208140 patients from 57 countries. Over a period of 1066572 person-years, 2046 KS cases were diagnosed. KS incidence rates per 100000 person-years were 52 in the Asia-Pacific and ranged between 180 and 280 in the other regions. KS risk was 5 times higher in South African women (aHR, 4.56; 95% confidence intervals [CI], 2.73-7.62) than in their European counterparts, and 2 times higher in South African men (2.21; 1.34-3.63). In Europe, Latin, and North America KS risk was 6 times higher in men who have sex with men (aHR, 5.95; 95% CI, 5.09-6.96) than in women. Comparing patients with current CD4 cell counts ≥700 cells/µL with those whose counts were
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- 2017
30. Post-traumatic stress disorder as a risk factor for major adverse cardiovascular events: a cohort study of a South African medical insurance scheme
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Mesa-Vieira, Cristina, primary, Didden, Christiane, additional, Schomaker, Michael, additional, Mouton, Johannes P., additional, Folb, Naomi, additional, van den Heuvel, Leigh L., additional, Gastaldon, Chiara, additional, Cornell, Morna, additional, Tlali, Mpho, additional, Kassanjee, Reshma, additional, Franco, Oscar H., additional, Seedat, Soraya, additional, and Haas, Andreas D., additional
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- 2024
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31. Bootstrap Inference when Using Multiple Imputation
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Schomaker, Michael and Heumann, Christian
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Statistics - Methodology - Abstract
Many modern estimators require bootstrapping to calculate confidence intervals because either no analytic standard error is available or the distribution of the parameter of interest is non-symmetric. It remains however unclear how to obtain valid bootstrap inference when dealing with multiple imputation to address missing data. We present four methods which are intuitively appealing, easy to implement, and combine bootstrap estimation with multiple imputation. We show that three of the four approaches yield valid inference, but that the performance of the methods varies with respect to the number of imputed data sets and the extent of missingness. Simulation studies reveal the behavior of our approaches in finite samples. A topical analysis from HIV treatment research, which determines the optimal timing of antiretroviral treatment initiation in young children, demonstrates the practical implications of the four methods in a sophisticated and realistic setting. This analysis suffers from missing data and uses the $g$-formula for inference, a method for which no standard errors are available.
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- 2016
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32. Estimating the effect of central bank independence on inflation using longitudinal targeted maximum likelihood estimation
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Baumann Philipp F. M., Schomaker Michael, and Rossi Enzo
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causal inference ,doubly robust ,super learning ,macroeconomics ,monetary policy ,62p20 ,Mathematics ,QA1-939 ,Probabilities. Mathematical statistics ,QA273-280 - Abstract
The notion that an independent central bank reduces a country’s inflation is a controversial hypothesis. To date, it has not been possible to satisfactorily answer this question because the complex macroeconomic structure that gives rise to the data has not been adequately incorporated into statistical analyses. We develop a causal model that summarizes the economic process of inflation. Based on this causal model and recent data, we discuss and identify the assumptions under which the effect of central bank independence on inflation can be identified and estimated. Given these and alternative assumptions, we estimate this effect using modern doubly robust effect estimators, i.e., longitudinal targeted maximum likelihood estimators. The estimation procedure incorporates machine learning algorithms and is tailored to address the challenges associated with complex longitudinal macroeconomic data. We do not find strong support for the hypothesis that having an independent central bank for a long period of time necessarily lowers inflation. Simulation studies evaluate the sensitivity of the proposed methods in complex settings when certain assumptions are violated and highlight the importance of working with appropriate learning algorithms for estimation.
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- 2021
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33. When and when not to use optimal model averaging
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Schomaker, Michael and Heumann, Christian
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- 2020
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34. The Effect of Electrical Load Shedding on Pediatric Hospital Admissions in South Africa
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Gehringer, Christian, Rode, Heinz, and Schomaker, Michael
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- 2018
35. HIV programmatic outcomes following implementation of the 'Treat-All' policy in a public sector setting in Eswatini: a prospective cohort study
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Kerschberger, Bernhard, Schomaker, Michael, Jobanputra, Kiran, Kabore, Serge M., Teck, Roger, Mabhena, Edwin, Mthethwa-Hleza, Simangele, Rusch, Barbara, Ciglenecki, Iza, and Boulle, Andrew
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Highly active antiretroviral therapy -- Analysis ,Patient compliance -- Analysis ,HIV -- Care and treatment ,Antiretroviral agents -- Analysis ,Public sector -- Analysis ,Health ,World Health Organization - Abstract
Introduction: The Treat-All policy--antiretroviral therapy (ART) initiation irrespective of CD4 cell criteria--increases access to treatment. Many ART programmes, however, reported increasing attrition and viral failure during treatment expansion, questioning the programmatic feasibility of Treat-All in resource-limited settings. We aimed to describe and compare programmatic outcomes between Treat-All and standard of care (SOC) in the public sectors of Eswatini. Methods: This is a prospective cohort study of [greater than or equal to]16-year-old HIV-positive patients initiated on first-line ART under Treat-Al and SOC in 18 health facilities of the Shiselweni region, from October 2014 to March 2016. SOC followed the CD4 350 and 500 cells/[mm.sup.3] treatment eligibility thresholds. Kaplan-Meier estimates were used to describe crude programmatic outcomes. Multivariate flexible parametric survival models were built to assess associations of time from ART initiation with the composite unfavourable outcome of all-cause attrition and viral failure. Results: Of the 3170 patients, 1888 (59.6%) initiated ART under Treat-All at a median CD4 cell count of 329 (IQR 168 to 488) cells/[mm.sup.3] compared with 292 (IQR 161 to 430) (p < 0.001) under SOC. Although crude programme retention at 36 months tended to be lower under Treat-All (71%) than SOC (75%) (p = 0.002), it was similar in covariate-adjusted analysis (adjusted hazard ratio [aHR] 1.06, 95% CI 0.91 to 1.23). The hazard of viral suppression was higher for Treat-All (aHR 1.12, 95% CI 1.01 to 1.23), while the hazard of viral failure was comparable (Treat-All: aHR 0.89, 95% CI 0.53 to 1.49). Among patients with advanced HIV disease (n = 1080), those under Treat-All (aHR 1.13, 95% CI 0.88 to 1.44) had a similar risk of an composite unfavourable outcome to SOC. Factors increasing the risk of the composite unfavourable outcome under both interventions were aged 16 to 24 years, being unmarried, anaemia, ART initiation on the same day as HIV care enrolment and CD4 [less than or equal to] 100 cells/[mm.sup.3]. Under Treat-All only, the risk of the unfavourable outcome was higher for pregnant women, WHO III/IV clinical stage and elevated creatinine. Conclusions: Compared to SOC, Treat-All resulted in comparable retention, improved viral suppression and comparable composite outcomes of retention without viral failure. Keywords: treat all; retention; viral failure; Swaziland; Eswatini; HIV, 1 | INTRODUCTION The World Health Organization (WHO) recommends antiretroviral therapy (ART) initiation at the time of HIV diagnosis irrespective of clinical and immunological criteria, aiming at improving patient-level outcomes [...]
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- 2020
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36. Stunting and growth velocity of adolescents with perinatally acquired HIV: differential evolution for males and females. A multiregional analysis from the IeDEA global paediatric collaboration
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Jesson, Julie, Schomaker, Michael, Malasteste, Karen, Wati, Dewi K., Kariminia, Azar, Sylla, Mariam, Kouadio, Kouakou, Sawry, Shobna, Mubiana-Mbewe, Mwangelwa, Ayaya, Samuel, Vreeman, Rachel, McGowan, Catherine C., Yotebieng, Marcel, Leroy, Valeriane, and Davies, Mary-Ann
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United States. National Institutes of Health -- Analysis ,United States. National Institute of Allergy and Infectious Diseases -- Analysis ,HIV -- Growth -- Analysis ,Antiretroviral agents -- Analysis ,Adolescence -- Analysis ,Company growth ,Company acquisition/merger ,Health ,World Health Organization -- Growth - Abstract
Introduction: Stunting is a key issue for adolescents with perinatally acquired HIV (APH) that needs to be better understood. As part of the IeDEA multiregional consortium, we described growth evolution during adolescence for APH on antiretroviral therapy (ART). Methods: We included data from sub-Saharan Africa, the Asia-Pacific, and the Caribbean, Central and South America regions collected between 2003 and 2016. Adolescents on ART, reporting perinatally acquired infection or entering HIV care before 10 years of age, with at least one height measurement between 10 and 16 years of age, and followed in care until at least 14 years of age were included. Characteristics at ART initiation and at 10 years of age were compared by sex. Correlates of growth defined by height-for-age z-scores (HAZ) between ages 10 and 19 years were studied separately for males and females, using linear mixed models. Results: Overall, 8737 APH were included, with 46% from Southern Africa. Median age at ART initiation was 8.1 years (interquartile range (IQR) 6.1 to 9.6), 50% were females, and 41% were stunted (HAZ Conclusions: Prevalence of stunting is high among APH worldwide. Substantial sex-based differences in growth evolution during adolescence were observed in this global cohort, which were not explained by differences in age of access to HIV care, degree of immunosuppression or region. Other factors influencing growth differences in APH, such as differences in puberta development, should be better documented, to guide further research and inform interventions to optimize growth and health outcomes among APH. Keywords: HIV; adolescent; growth; stunting; cohort studies; developing countries, 1 | INTRODUCTION Adolescence, defined by the World Health Organization (WHO) as between 10 and 19 years of age [1], is a critical transition period in life, accompanied by significant [...]
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- 2019
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37. Feasibility of antiretroviral therapy initiation under the treat-all policy under routine conditions: a prospective cohort study from Eswatini
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Kerschberger, Bernhard, Jobanputra, Kiran, Schomaker, Michael, Kabore, Serge M., Teck, Roger, Mabhena, Edwin, Lukhele, Nomthandazo, Rusch, Barbara, Boulle, Andrew, and Ciglenecki, Iza
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HIV patients -- Comparative analysis ,Highly active antiretroviral therapy -- Comparative analysis ,HIV -- Comparative analysis ,Medical research -- Comparative analysis ,Antiretroviral agents -- Comparative analysis ,Tuberculosis -- Comparative analysis ,Pregnant women -- Comparative analysis ,Health ,World Health Organization - Abstract
Introduction: The World Health Organization recommends the Treat-All policy of immediate antiretroviral therapy (ART) initiation, but questions persist about its feasibility in resource-poor settings. We assessed the feasibility of Treat-All compared with standard of care (SOC) under routine conditions. Methods: This prospective cohort study from southern Eswatini followed adults from HIV care enrolment to ART initiation. Between October 2014 and March 2016, Treat-All was offered in one health zone and SOC according to the CD4 350 and 500 cells/[mm.sup.3] treatment eligibility thresholds in the neighbouring health zone, each of which comprised one secondary and eight primary care facilities. We used Kaplan--Meier estimates, multivariate flexible parametric survival models and standardized survival curves to compare ART initiation between the two interventions. Results: Of the 1726 (57.3%) patients enrolled under Treat-All and 1287 (42.7%) under SOC, cumulative three-month ART initiation was higher under Treat-All (91%) than SOC (74%; p < 0.001) with a median time to ART of 1 (IQR 0 to 14) and 10 (IQR 2 to 117) days respectively. Under Treat-All, ART initiation was higher in pregnant women (vs. non-pregnant women: adjusted hazard ratio (aHR) 1.96, 95% confidence interval (CI) 1.70 to 2.26), those with secondary education (vs. no formal education: aHR 1.48, 95% CI 1.12 to 1.95), and patients with an HIV-positive diagnosis before care enrolment (aHR 1.22, 95% CI 1.10 to 1.36). ART initiation was lower in patients attending secondary care facilities (aHR 0.64, 95% CI 0.58 to 0.72) and for CD4 351 to 500 when compared with CD4 201 to 350 cells/[mm.sup.3] (aHR 0.84, 95% CI 0.72 to 1.00). ART initiation varied over time for TB cases, with lower hazard during the first two weeks after HIV care enrolment and higher hazards thereafter. Of patients with advanced HIV disease (n = 1085; 36.0%), crude 3-month ART initiation was similar in both interventions (91% to 92%) although Treat-All initiated patients more quickly during the first month after HIV care enrolment. Conclusions: ART initiation was high under Treat-All and without evidence of de-prioritization of patients with advanced HIV disease. Additional studies are needed to understand the long-term impact of Treat-All on patient outcomes. Keywords: treat all; ART initiation; linkage; Eswatini; universal ART; treatment cascade, 1 | INTRODUCTION The therapeutic effect of antiretroviral therapy (ART) and its preventive benefits of reducing HIV transmission are well established [1-3]. On the basis of this evidence, the World [...]
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- 2019
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38. Virologic response of adolescents living with perinatally acquired HIV receiving antiretroviral therapy in the period of early adolescence (10–14 years) in South Africa
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Nyakato, Patience, Schomaker, Michael, Sipambo, Nosisa, Technau, Karl-Günter, Fatti, Geoffrey, Rabie, Helena, Tanser, Frank, Eley, Brian, Euvrard, Jonathan, Wood, Robin, Tsondai, Priscilla R., Yiannoutsos, Constantin T., Cornell, Morna, and Davies, Mary-Ann
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- 2021
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39. Inference
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2016
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40. Association of Two Variables
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2016
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41. Hypothesis Testing
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2016
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42. Linear Regression
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2016
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43. Random Variables
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2016
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44. Combinatorics
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2016
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45. Elements of Probability Theory
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2016
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46. Probability Distributions
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2016
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47. Measures of Central Tendency and Dispersion
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2016
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48. Introduction and Framework
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2016
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49. Frequency Measures and Graphical Representation of Data
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Heumann, Christian, Schomaker, Michael, Shalabh, Heumann, Christian, Schomaker, Michael, and Shalabh
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- 2016
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50. Targeted Maximum Likelihood Estimation for Dynamic and Static Longitudinal Marginal Structural Working Models
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Petersen, Maya, Schwab, Joshua, Gruber, Susan, Blaser, Nello, Schomaker, Michael, and van der Laan, Mark
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HIV/AIDS ,dynamic regime ,semiparametric statistical model ,targeted minimum loss based estimation ,confounding ,right censoring - Abstract
This paper describes a targeted maximum likelihood estimator (TMLE) for the parameters of longitudinal static and dynamic marginal structural models. We consider a longitudinal data structure consisting of baseline covariates, time-dependent intervention nodes, intermediate time-dependent covariates, and a possibly time-dependent outcome. The intervention nodes at each time point can include a binary treatment as well as a right-censoring indicator. Given a class of dynamic or static interventions, a marginal structural model is used to model the mean of the intervention-specific counterfactual outcome as a function of the intervention, time point, and possibly a subset of baseline covariates. Because the true shape of this function is rarely known, the marginal structural model is used as a working model. The causal quantity of interest is defined as the projection of the true function onto this working model. Iterated conditional expectation double robust estimators for marginal structural model parameters were previously proposed by Robins (2000, 2002) and Bang and Robins (2005). Here we build on this work and present a pooled TMLE for the parameters of marginal structural working models. We compare this pooled estimator to a stratified TMLE (Schnitzer et al. 2014) that is based on estimating the intervention-specific mean separately for each intervention of interest. The performance of the pooled TMLE is compared to the performance of the stratified TMLE and the performance of inverse probability weighted (IPW) estimators using simulations. Concepts are illustrated using an example in which the aim is to estimate the causal effect of delayed switch following immunological failure of first line antiretroviral therapy among HIV-infected patients. Data from the International Epidemiological Databases to Evaluate AIDS, Southern Africa are analyzed to investigate this question using both TML and IPW estimators. Our results demonstrate practical advantages of the pooled TMLE over an IPW estimator for working marginal structural models for survival, as well as cases in which the pooled TMLE is superior to its stratified counterpart.
- Published
- 2014
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