{"title":"有竞争风险的观察性研究中平均治疗效果的基于重采样的置信区间和带宽","authors":"Jasmin Rühl, Sarah Friedrich","doi":"10.1007/s11222-024-10420-w","DOIUrl":null,"url":null,"abstract":"<p>The g-formula can be used to estimate the treatment effect while accounting for confounding bias in observational studies. With regard to time-to-event endpoints, possibly subject to competing risks, the construction of valid pointwise confidence intervals and time-simultaneous confidence bands for the causal risk difference is complicated, however. A convenient solution is to approximate the asymptotic distribution of the corresponding stochastic process by means of resampling approaches. In this paper, we consider three different resampling methods, namely the classical nonparametric bootstrap, the influence function equipped with a resampling approach as well as a martingale-based bootstrap version, the so-called wild bootstrap. For the latter, three sub-versions based on differing distributions of the underlying random multipliers are examined. We set up a simulation study to compare the accuracy of the different techniques, which reveals that the wild bootstrap should in general be preferred if the sample size is moderate and sufficient data on the event of interest have been accrued. For illustration, the resampling methods are further applied to data on the long-term survival in patients with early-stage Hodgkin’s disease.</p>","PeriodicalId":22058,"journal":{"name":"Statistics and Computing","volume":null,"pages":null},"PeriodicalIF":1.6000,"publicationDate":"2024-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Resampling-based confidence intervals and bands for the average treatment effect in observational studies with competing risks\",\"authors\":\"Jasmin Rühl, Sarah Friedrich\",\"doi\":\"10.1007/s11222-024-10420-w\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p>The g-formula can be used to estimate the treatment effect while accounting for confounding bias in observational studies. With regard to time-to-event endpoints, possibly subject to competing risks, the construction of valid pointwise confidence intervals and time-simultaneous confidence bands for the causal risk difference is complicated, however. A convenient solution is to approximate the asymptotic distribution of the corresponding stochastic process by means of resampling approaches. In this paper, we consider three different resampling methods, namely the classical nonparametric bootstrap, the influence function equipped with a resampling approach as well as a martingale-based bootstrap version, the so-called wild bootstrap. For the latter, three sub-versions based on differing distributions of the underlying random multipliers are examined. We set up a simulation study to compare the accuracy of the different techniques, which reveals that the wild bootstrap should in general be preferred if the sample size is moderate and sufficient data on the event of interest have been accrued. For illustration, the resampling methods are further applied to data on the long-term survival in patients with early-stage Hodgkin’s disease.</p>\",\"PeriodicalId\":22058,\"journal\":{\"name\":\"Statistics and Computing\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":1.6000,\"publicationDate\":\"2024-03-21\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Statistics and Computing\",\"FirstCategoryId\":\"100\",\"ListUrlMain\":\"https://doi.org/10.1007/s11222-024-10420-w\",\"RegionNum\":2,\"RegionCategory\":\"数学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"COMPUTER SCIENCE, THEORY & METHODS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Statistics and Computing","FirstCategoryId":"100","ListUrlMain":"https://doi.org/10.1007/s11222-024-10420-w","RegionNum":2,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"COMPUTER SCIENCE, THEORY & METHODS","Score":null,"Total":0}
引用次数: 0
摘要
g 公式可用于估计治疗效果,同时考虑观察性研究中的混杂偏倚。然而,对于可能存在竞争风险的时间到事件终点,构建有效的因果风险差异点式置信区间和时间同步置信区间非常复杂。一个方便的解决方案是通过重采样方法来近似相应随机过程的渐近分布。在本文中,我们考虑了三种不同的重采样方法,即经典的非参数自举法、配备重采样方法的影响函数以及基于马氏自举法的版本,即所谓的野生自举法。对于后者,我们研究了基于基础随机乘数不同分布的三个子版本。我们建立了一个模拟研究来比较不同技术的准确性,结果表明,如果样本量适中,并且已经积累了足够的相关事件的数据,一般情况下野生自举法更受欢迎。为了说明问题,我们还将重采样方法进一步应用于早期霍奇金病患者的长期生存数据。
Resampling-based confidence intervals and bands for the average treatment effect in observational studies with competing risks
The g-formula can be used to estimate the treatment effect while accounting for confounding bias in observational studies. With regard to time-to-event endpoints, possibly subject to competing risks, the construction of valid pointwise confidence intervals and time-simultaneous confidence bands for the causal risk difference is complicated, however. A convenient solution is to approximate the asymptotic distribution of the corresponding stochastic process by means of resampling approaches. In this paper, we consider three different resampling methods, namely the classical nonparametric bootstrap, the influence function equipped with a resampling approach as well as a martingale-based bootstrap version, the so-called wild bootstrap. For the latter, three sub-versions based on differing distributions of the underlying random multipliers are examined. We set up a simulation study to compare the accuracy of the different techniques, which reveals that the wild bootstrap should in general be preferred if the sample size is moderate and sufficient data on the event of interest have been accrued. For illustration, the resampling methods are further applied to data on the long-term survival in patients with early-stage Hodgkin’s disease.
期刊介绍:
Statistics and Computing is a bi-monthly refereed journal which publishes papers covering the range of the interface between the statistical and computing sciences.
In particular, it addresses the use of statistical concepts in computing science, for example in machine learning, computer vision and data analytics, as well as the use of computers in data modelling, prediction and analysis. Specific topics which are covered include: techniques for evaluating analytically intractable problems such as bootstrap resampling, Markov chain Monte Carlo, sequential Monte Carlo, approximate Bayesian computation, search and optimization methods, stochastic simulation and Monte Carlo, graphics, computer environments, statistical approaches to software errors, information retrieval, machine learning, statistics of databases and database technology, huge data sets and big data analytics, computer algebra, graphical models, image processing, tomography, inverse problems and uncertainty quantification.
In addition, the journal contains original research reports, authoritative review papers, discussed papers, and occasional special issues on particular topics or carrying proceedings of relevant conferences. Statistics and Computing also publishes book review and software review sections.