Ilaria Prosepe, Saskia le Cessie, Hein Putter, Nan van Geloven
{"title":"Causal Multistate Models to Evaluate Treatment Delay.","authors":"Ilaria Prosepe, Saskia le Cessie, Hein Putter, Nan van Geloven","doi":"10.1002/sim.70061","DOIUrl":null,"url":null,"abstract":"<p><p>Multistate models allow for the study of scenarios where individuals experience different events over time. While effective for descriptive and predictive purposes, multistate models are not typically used for causal inference. We propose an estimator that combines a multistate model with g-computation to estimate the causal effect of treatment delay strategies. In particular, we estimate the impact of strategies such as awaiting natural recovery for 3 months, on the marginal probability of recovery. We use an illness-death model, where illness and death represent, respectively, treatment and recovery. We formulate the causal assumptions needed for identification and the modeling assumptions needed to estimate the quantities of interest. In a simulation study, we present scenarios where the proposed method can make more efficient use of data compared to an alternative approach using cloning-censoring-reweighting. We then showcase the proposed methodology on real data by estimating the effect of treatment delay on a cohort of 1896 couples with unexplained subfertility who seek intrauterine insemination.</p>","PeriodicalId":21879,"journal":{"name":"Statistics in Medicine","volume":"44 7","pages":"e70061"},"PeriodicalIF":1.8000,"publicationDate":"2025-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Statistics in Medicine","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/sim.70061","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"MATHEMATICAL & COMPUTATIONAL BIOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Multistate models allow for the study of scenarios where individuals experience different events over time. While effective for descriptive and predictive purposes, multistate models are not typically used for causal inference. We propose an estimator that combines a multistate model with g-computation to estimate the causal effect of treatment delay strategies. In particular, we estimate the impact of strategies such as awaiting natural recovery for 3 months, on the marginal probability of recovery. We use an illness-death model, where illness and death represent, respectively, treatment and recovery. We formulate the causal assumptions needed for identification and the modeling assumptions needed to estimate the quantities of interest. In a simulation study, we present scenarios where the proposed method can make more efficient use of data compared to an alternative approach using cloning-censoring-reweighting. We then showcase the proposed methodology on real data by estimating the effect of treatment delay on a cohort of 1896 couples with unexplained subfertility who seek intrauterine insemination.
期刊介绍:
The journal aims to influence practice in medicine and its associated sciences through the publication of papers on statistical and other quantitative methods. Papers will explain new methods and demonstrate their application, preferably through a substantive, real, motivating example or a comprehensive evaluation based on an illustrative example. Alternatively, papers will report on case-studies where creative use or technical generalizations of established methodology is directed towards a substantive application. Reviews of, and tutorials on, general topics relevant to the application of statistics to medicine will also be published. The main criteria for publication are appropriateness of the statistical methods to a particular medical problem and clarity of exposition. Papers with primarily mathematical content will be excluded. The journal aims to enhance communication between statisticians, clinicians and medical researchers.