Statistics in Medicine最新文献

{"title":"Estimating Time-Varying Exposure Effects Through Continuous-Time Modelling in Mendelian Randomization.","authors":"Haodong Tian, Ashish Patel, Stephen Burgess","doi":"10.1002/sim.10222","DOIUrl":"https://doi.org/10.1002/sim.10222","url":null,"abstract":"<p><p>Mendelian randomization is an instrumental variable method that utilizes genetic information to investigate the causal effect of a modifiable exposure on an outcome. In most cases, the exposure changes over time. Understanding the time-varying causal effect of the exposure can yield detailed insights into mechanistic effects and the potential impact of public health interventions. Recently, a growing number of Mendelian randomization studies have attempted to explore time-varying causal effects. However, the proposed approaches oversimplify temporal information and rely on overly restrictive structural assumptions, limiting their reliability in addressing time-varying causal problems. This article considers a novel approach to estimate time-varying effects through continuous-time modelling by combining functional principal component analysis and weak-instrument-robust techniques. Our method effectively utilizes available data without making strong structural assumptions and can be applied in general settings where the exposure measurements occur at different timepoints for different individuals. We demonstrate through simulations that our proposed method performs well in estimating time-varying effects and provides reliable inference when the time-varying effect form is correctly specified. The method could theoretically be used to estimate arbitrarily complex time-varying effects. However, there is a trade-off between model complexity and instrument strength. Estimating complex time-varying effects requires instruments that are unrealistically strong. We illustrate the application of this method in a case study examining the time-varying effects of systolic blood pressure on urea levels.</p>","PeriodicalId":21879,"journal":{"name":"Statistics in Medicine","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142381657","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Regression Approaches to Assess Effect of Treatments That Arrest Progression of Symptoms.","authors":"Ana M Ortega-Villa, Martha C Nason, Michael P Fay, Sara Alehashemi, Raphaela Goldbach-Mansky, Dean A Follmann","doi":"10.1002/sim.10219","DOIUrl":"https://doi.org/10.1002/sim.10219","url":null,"abstract":"<p><p>Motivated by a small sample example in neonatal onset multisystem inflammatory disease (NOMID), we propose a method that can be used when the interest is testing for an association between a changes in disease progression with start of treatment compared to historical disease progression prior to treatment. Our method estimates the longitudinal trajectory of the outcome variable and adds an interaction term between an intervention indicator variable and the time since initiation of the intervention. This method is appropriate for a situation in which the intervention slows or arrests the effect of the disease on the outcome, as is the case in our motivating example. By simulation in small samples and restricted sets of treatment initiation times, we show that the generalized estimating equations (GEE) formulation with small sample adjustments can bound the Type I error rate better than GEE and linear mixed models without small sample adjustments. Permutation tests (permuting the time of treatment initiation) is another valid approach that can also be useful. We illustrate the methodology through an application to a prospective cohort of NOMID patients enrolled at the NIH clinical center.</p>","PeriodicalId":21879,"journal":{"name":"Statistics in Medicine","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142372927","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Latent Archetypes of the Spatial Patterns of Cancer.","authors":"Thaís Pacheco Menezes, Marcos Oliveira Prates, Renato Assunção, Mônica Silva Monteiro De Castro","doi":"10.1002/sim.10232","DOIUrl":"https://doi.org/10.1002/sim.10232","url":null,"abstract":"<p><p>The cancer atlas edited by several countries is the main resource for the analysis of the geographic variation of cancer risk. Correlating the observed spatial patterns with known or hypothesized risk factors is time-consuming work for epidemiologists who need to deal with each cancer separately, breaking down the patterns according to sex and race. The recent literature has proposed to study more than one cancer simultaneously looking for common spatial risk factors. However, this previous work has two constraints: they consider only a very small (2-4) number of cancers previously known to share risk factors. In this article, we propose an exploratory method to search for latent spatial risk factors of a large number of supposedly unrelated cancers. The method is based on the singular value decomposition and nonnegative matrix factorization, it is computationally efficient, scaling easily with the number of regions and cancers. We carried out a simulation study to evaluate the method's performance and apply it to cancer atlas from the USA, England, France, Australia, Spain, and Brazil. We conclude that with very few latent maps, which can represent a reduction of up to 90% of atlas maps, most of the spatial variability is conserved. By concentrating on the epidemiological analysis of these few latent maps a substantial amount of work is saved and, at the same time, high-level explanations affecting many cancers simultaneously can be reached.</p>","PeriodicalId":21879,"journal":{"name":"Statistics in Medicine","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142372925","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pairwise Accelerated Failure Time Regression Models for Infectious Disease Transmission in Close-Contact Groups With External Sources of Infection.","authors":"Yushuf Sharker, Zaynab Diallo, Wasiur R KhudaBukhsh, Eben Kenah","doi":"10.1002/sim.10226","DOIUrl":"https://doi.org/10.1002/sim.10226","url":null,"abstract":"<p><p>Many important questions in infectious disease epidemiology involve associations between covariates (e.g., age or vaccination status) and infectiousness or susceptibility. Because disease transmission produces dependent outcomes, these questions are difficult or impossible to address using standard regression models from biostatistics. Pairwise survival analysis handles dependent outcomes by calculating likelihoods in terms of contact interval distributions in ordered pairs of individuals. The contact interval in the ordered pair <math> <semantics><mrow><mi>i</mi> <mi>j</mi></mrow> <annotation>$$ ij $$</annotation></semantics> </math> is the time from the onset of infectiousness in <math> <semantics><mrow><mi>i</mi></mrow> <annotation>$$ i $$</annotation></semantics> </math> to infectious contact from <math> <semantics><mrow><mi>i</mi></mrow> <annotation>$$ i $$</annotation></semantics> </math> to <math> <semantics><mrow><mi>j</mi></mrow> <annotation>$$ j $$</annotation></semantics> </math> , where an infectious contact is sufficient to infect <math> <semantics><mrow><mi>j</mi></mrow> <annotation>$$ j $$</annotation></semantics> </math> if they are susceptible. Here, we introduce a pairwise accelerated failure time regression model for infectious disease transmission that allows the rate parameter of the contact interval distribution to depend on individual-level infectiousness covariates for <math> <semantics><mrow><mi>i</mi></mrow> <annotation>$$ i $$</annotation></semantics> </math> , individual-level susceptibility covariates for <math> <semantics><mrow><mi>j</mi></mrow> <annotation>$$ j $$</annotation></semantics> </math> , and pair-level covariates (e.g., type of relationship). This model can simultaneously handle internal infections (caused by transmission between individuals under observation) and external infections (caused by environmental or community sources of infection). We show that this model produces consistent and asymptotically normal parameter estimates. In a simulation study, we evaluate bias and confidence interval coverage probabilities, explore the role of epidemiologic study design, and investigate the effects of model misspecification. We use this regression model to analyze household data from Los Angeles County during the 2009 influenza A (H1N1) pandemic, where we find that the ability to account for external sources of infection increases the statistical power to estimate the effect of antiviral prophylaxis.</p>","PeriodicalId":21879,"journal":{"name":"Statistics in Medicine","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142372926","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Weighted Expectile Regression Neural Networks for Right Censored Data.","authors":"Feipeng Zhang, Xi Chen, Peng Liu, Caiyun Fan","doi":"10.1002/sim.10221","DOIUrl":"https://doi.org/10.1002/sim.10221","url":null,"abstract":"<p><p>As a favorable alternative to the censored quantile regression, censored expectile regression has been popular in survival analysis due to its flexibility in modeling the heterogeneous effect of covariates. The existing weighted expectile regression (WER) method assumes that the censoring variable and covariates are independent, and that the covariates effects has a global linear structure. However, these two assumptions are too restrictive to capture the complex and nonlinear pattern of the underlying covariates effects. In this article, we developed a novel weighted expectile regression neural networks (WERNN) method by incorporating the deep neural network structure into the censored expectile regression framework. To handle the random censoring, we employ the inverse probability of censoring weighting (IPCW) technique in the expectile loss function. The proposed WERNN method is flexible enough to fit nonlinear patterns and therefore achieves more accurate prediction performance than the existing WER method for right censored data. Our findings are supported by extensive Monte Carlo simulation studies and a real data application.</p>","PeriodicalId":21879,"journal":{"name":"Statistics in Medicine","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354091","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prefiltered component-based greedy (PreCoG) scan method.","authors":"Joshua P French, Mohammad Meysami, Ettie M Lipner","doi":"10.1002/sim.10170","DOIUrl":"10.1002/sim.10170","url":null,"abstract":"<p><p>The spatial distribution of disease cases can provide important insights into disease spread and its potential risk factors. Identifying disease clusters correctly can help us discover new risk factors and inform interventions to control and prevent the spread of disease as quickly as possible. In this study, we propose a novel scan method, the Prefiltered Component-based Greedy (PreCoG) scan method, which efficiently and accurately detects irregularly shaped clusters using a prefiltered component-based algorithm. The PreCoG scan method's flexibility allows it to perform well in detecting both regularly and irregularly-shaped clusters. Additionally, it is fast to apply while providing high power, sensitivity, and positive predictive value for the detected clusters compared to other scan methods. To confirm the effectiveness of the PreCoG method, we compare its performance to many other scan methods. Additionally, we have implemented this method in the smerc R package to make it publicly available to other researchers. Our proposed PreCoG scan method presents a unique and innovative process for detecting disease clusters and can improve the accuracy of disease surveillance systems.</p>","PeriodicalId":21879,"journal":{"name":"Statistics in Medicine","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141591382","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Bayesian modeling of spatial ordinal data from health surveys.","authors":"Miguel Ángel Beltrán-Sánchez, Miguel-Angel Martinez-Beneito, Ana Corberán-Vallet","doi":"10.1002/sim.10166","DOIUrl":"10.1002/sim.10166","url":null,"abstract":"<p><p>Health surveys allow exploring health indicators that are of great value from a public health point of view and that cannot normally be studied from regular health registries. These indicators are usually coded as ordinal variables and may depend on covariates associated with individuals. In this article, we propose a Bayesian individual-level model for small-area estimation of survey-based health indicators. A categorical likelihood is used at the first level of the model hierarchy to describe the ordinal data, and spatial dependence among small areas is taken into account by using a conditional autoregressive distribution. Post-stratification of the results of the proposed individual-level model allows extrapolating the results to any administrative areal division, even for small areas. We apply this methodology to describe the geographical distribution of a self-perceived health indicator from the Health Survey of the Region of Valencia (Spain) for the year 2016.</p>","PeriodicalId":21879,"journal":{"name":"Statistics in Medicine","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141634608","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An augmented illness-death model for semi-competing risks with clinically immediate terminal events.","authors":"Harrison T Reeder, Kyu Ha Lee, Stefania I Papatheodorou, Sebastien Haneuse","doi":"10.1002/sim.10181","DOIUrl":"10.1002/sim.10181","url":null,"abstract":"<p><p>Preeclampsia is a pregnancy-associated condition posing risks of both fetal and maternal mortality and morbidity that can only resolve following delivery and removal of the placenta. Because in its typical form preeclampsia can arise before delivery, but not after, these two events exemplify the time-to-event setting of \"semi-competing risks\" in which a non-terminal event of interest is subject to the occurrence of a terminal event of interest. The semi-competing risks framework presents a valuable opportunity to simultaneously address two clinically meaningful risk modeling tasks: (i) characterizing risk of developing preeclampsia, and (ii) characterizing time to delivery after onset of preeclampsia. However, some people with preeclampsia deliver immediately upon diagnosis, while others are admitted and monitored for an extended period before giving birth, resulting in two distinct trajectories following the non-terminal event, which we call \"clinically immediate\" and \"non-immediate\" terminal events. Though such phenomena arise in many clinical contexts, to-date there have not been methods developed to acknowledge the complex dependencies between such outcomes, nor leverage these phenomena to gain new insight into individualized risk. We address this gap by proposing a novel augmented frailty-based illness-death model with a binary submodel to distinguish risk of immediate terminal event following the non-terminal event. The model admits direct dependence of the terminal event on the non-terminal event through flexible regression specification, as well as indirect dependence via a shared frailty term linking each submodel. We develop an efficient Bayesian sampler for estimation and corresponding model fit metrics, and derive formulae for dynamic risk prediction. In an extended example using pregnancy outcome data from an electronic health record, we demonstrate the proposed model's direct applicability to address a broad range of clinical questions.</p>","PeriodicalId":21879,"journal":{"name":"Statistics in Medicine","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141749119","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Covariate-adjusted generalized pairwise comparisons in small samples.","authors":"Stijn Jaspers, Johan Verbeeck, Olivier Thas","doi":"10.1002/sim.10140","DOIUrl":"10.1002/sim.10140","url":null,"abstract":"<p><p>Semiparametric probabilistic index models allow for the comparison of two groups of observations, whilst adjusting for covariates, thereby fitting nicely within the framework of generalized pairwise comparisons (GPC). As with most regression approaches in this setting, the limited amount of data results in invalid inference as the asymptotic normality assumption is not met. In addition, separation issues might arise when considering small samples. In this article, we show that the parameters of the probabilistic index model can be estimated using generalized estimating equations, for which adjustments exist that lead to estimators of the sandwich variance-covariance matrix with improved finite sample properties and that can deal with bias due to separation. In this way, appropriate inference can be performed as is shown through extensive simulation studies. The known relationships between the probabilistic index and other GPC statistics allow to also provide valid inference for example, the net treatment benefit or the success odds.</p>","PeriodicalId":21879,"journal":{"name":"Statistics in Medicine","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141498979","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A latent variable approach to jointly modeling longitudinal and cumulative event data using a weighted two-stage method.","authors":"Madeline R Abbott, Inbal Nahum-Shani, Cho Y Lam, Lindsey N Potter, David W Wetter, Walter H Dempsey","doi":"10.1002/sim.10171","DOIUrl":"10.1002/sim.10171","url":null,"abstract":"<p><p>Ecological momentary assessment (EMA), a data collection method commonly employed in mHealth studies, allows for repeated real-time sampling of individuals' psychological, behavioral, and contextual states. Due to the frequent measurements, data collected using EMA are useful for understanding both the temporal dynamics in individuals' states and how these states relate to adverse health events. Motivated by data from a smoking cessation study, we propose a joint model for analyzing longitudinal EMA data to determine whether certain latent psychological states are associated with repeated cigarette use. Our method consists of a longitudinal submodel-a dynamic factor model-that models changes in the time-varying latent states and a cumulative risk submodel-a Poisson regression model-that connects the latent states with the total number of events. In the motivating data, both the predictors-the underlying psychological states-and the event outcome-the number of cigarettes smoked-are partially unobservable; we account for this incomplete information in our proposed model and estimation method. We take a two-stage approach to estimation that leverages existing software and uses importance sampling-based weights to reduce potential bias. We demonstrate that these weights are effective at reducing bias in the cumulative risk submodel parameters via simulation. We apply our method to a subset of data from a smoking cessation study to assess the association between psychological state and cigarette smoking. The analysis shows that above-average intensities of negative mood are associated with increased cigarette use.</p>","PeriodicalId":21879,"journal":{"name":"Statistics in Medicine","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11338709/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141731416","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}