{"title":"用贝叶斯方法估算死亡截断的观察性研究中的不良反应。","authors":"Anthony Sisti, Andrew Zullo, Roee Gutman","doi":"10.1177/09622802241283170","DOIUrl":null,"url":null,"abstract":"<p><p>Death among subjects is common in observational studies evaluating the causal effects of interventions among geriatric or severely ill patients. High mortality rates complicate the comparison of the prevalence of adverse events between interventions. This problem is often referred to as outcome \"truncation\" by death. A possible solution is to estimate the survivor average causal effect, an estimand that evaluates the effects of interventions among those who would have survived under both treatment assignments. However, because the survivor average causal effect does not include subjects who would have died under one or both arms, it does not consider the relationship between adverse events and death. We propose a Bayesian method which imputes the unobserved mortality and adverse event outcomes for each participant under the intervention they did not receive. Using the imputed outcomes we define a composite ordinal outcome for each patient, combining the occurrence of death and the adverse event in an increasing scale of severity. This allows for the comparison of the effects of the interventions on death and the adverse event simultaneously among the entire sample. We implement the procedure to analyze the incidence of heart failure among geriatric patients being treated for Type II diabetes with sulfonylureas or dipeptidyl peptidase-4 inhibitors.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"2079-2097"},"PeriodicalIF":1.6000,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"A Bayesian method for adverse effects estimation in observational studies with truncation by death.\",\"authors\":\"Anthony Sisti, Andrew Zullo, Roee Gutman\",\"doi\":\"10.1177/09622802241283170\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Death among subjects is common in observational studies evaluating the causal effects of interventions among geriatric or severely ill patients. High mortality rates complicate the comparison of the prevalence of adverse events between interventions. This problem is often referred to as outcome \\\"truncation\\\" by death. A possible solution is to estimate the survivor average causal effect, an estimand that evaluates the effects of interventions among those who would have survived under both treatment assignments. However, because the survivor average causal effect does not include subjects who would have died under one or both arms, it does not consider the relationship between adverse events and death. We propose a Bayesian method which imputes the unobserved mortality and adverse event outcomes for each participant under the intervention they did not receive. Using the imputed outcomes we define a composite ordinal outcome for each patient, combining the occurrence of death and the adverse event in an increasing scale of severity. This allows for the comparison of the effects of the interventions on death and the adverse event simultaneously among the entire sample. We implement the procedure to analyze the incidence of heart failure among geriatric patients being treated for Type II diabetes with sulfonylureas or dipeptidyl peptidase-4 inhibitors.</p>\",\"PeriodicalId\":22038,\"journal\":{\"name\":\"Statistical Methods in Medical Research\",\"volume\":\" \",\"pages\":\"2079-2097\"},\"PeriodicalIF\":1.6000,\"publicationDate\":\"2024-11-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Statistical Methods in Medical Research\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1177/09622802241283170\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/11/5 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q3\",\"JCRName\":\"HEALTH CARE SCIENCES & SERVICES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Statistical Methods in Medical Research","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/09622802241283170","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/11/5 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
引用次数: 0
摘要
在评估干预措施对老年病人或重病患者的因果影响的观察性研究中,受试者死亡的情况很常见。高死亡率使比较不同干预措施的不良事件发生率变得更加复杂。这个问题通常被称为死亡导致的结果 "截断"。一种可能的解决方案是估算幸存者平均因果效应,这种估算方法可以评估干预措施对两种治疗方案下存活者的影响。然而,由于幸存者平均因果效应不包括在一种或两种治疗方法下都会死亡的受试者,因此它没有考虑不良事件与死亡之间的关系。我们提出了一种贝叶斯方法,该方法可估算出每位受试者在未接受干预的情况下未观察到的死亡率和不良事件结果。利用估算的结果,我们为每位患者定义了一个综合的序数结果,将死亡和不良事件的发生按严重程度递增结合起来。这样就可以在整个样本中同时比较干预措施对死亡和不良事件的影响。我们采用该方法分析了接受磺脲类药物或二肽基肽酶-4 抑制剂治疗的 II 型糖尿病老年患者的心力衰竭发生率。
A Bayesian method for adverse effects estimation in observational studies with truncation by death.
Death among subjects is common in observational studies evaluating the causal effects of interventions among geriatric or severely ill patients. High mortality rates complicate the comparison of the prevalence of adverse events between interventions. This problem is often referred to as outcome "truncation" by death. A possible solution is to estimate the survivor average causal effect, an estimand that evaluates the effects of interventions among those who would have survived under both treatment assignments. However, because the survivor average causal effect does not include subjects who would have died under one or both arms, it does not consider the relationship between adverse events and death. We propose a Bayesian method which imputes the unobserved mortality and adverse event outcomes for each participant under the intervention they did not receive. Using the imputed outcomes we define a composite ordinal outcome for each patient, combining the occurrence of death and the adverse event in an increasing scale of severity. This allows for the comparison of the effects of the interventions on death and the adverse event simultaneously among the entire sample. We implement the procedure to analyze the incidence of heart failure among geriatric patients being treated for Type II diabetes with sulfonylureas or dipeptidyl peptidase-4 inhibitors.
期刊介绍:
Statistical Methods in Medical Research is a peer reviewed scholarly journal and is the leading vehicle for articles in all the main areas of medical statistics and an essential reference for all medical statisticians. This unique journal is devoted solely to statistics and medicine and aims to keep professionals abreast of the many powerful statistical techniques now available to the medical profession. This journal is a member of the Committee on Publication Ethics (COPE)