{"title":"一种新的分层分析方法,用于测试和估计总体治疗对事件发生时间结果的影响,使用平均风险和生存体重。","authors":"Zihan Qian, Lu Tian, Miki Horiguchi, Hajime Uno","doi":"10.1002/sim.70056","DOIUrl":null,"url":null,"abstract":"<p><p>Given the limitations of using the Cox hazard ratio to summarize the magnitude of the treatment effect, alternative measures that do not have these limitations are gaining attention. One of the recently proposed alternative methods uses the average hazard with survival weight (AH). This population quantity can be interpreted as the average intensity of the event occurrence in a given time window that does not involve study-specific censoring. Inference procedures for the ratio of AH and difference in AH have already been proposed in simple randomized controlled trial settings to compare two groups. However, methods with stratification factors have not been well discussed, although stratified analysis is often used in practice to adjust for confounding factors and increase the power to detect a between-group difference. The conventional stratified analysis or meta-analysis approach, which integrates stratum-specific treatment effects using an optimal weight, directly applies to the ratio of AH and difference in AH. However, this conventional approach has significant limitations similar to the Cochran-Mantel-Haenszel method for a binary outcome and the stratified Cox procedure for a time-to-event outcome. To address this, we propose a new stratified analysis method for AH using standardization. With the proposed method, one can summarize the between-group treatment effect in both absolute difference and relative terms, adjusting for stratification factors. This can be a valuable alternative to the traditional stratified Cox procedure to estimate and report the magnitude of the treatment effect on time-to-event outcomes using hazard.</p>","PeriodicalId":21879,"journal":{"name":"Statistics in Medicine","volume":"44 7","pages":"e70056"},"PeriodicalIF":1.8000,"publicationDate":"2025-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"A Novel Stratified Analysis Method for Testing and Estimating Overall Treatment Effects on Time-To-Event Outcomes Using Average Hazard With Survival Weight.\",\"authors\":\"Zihan Qian, Lu Tian, Miki Horiguchi, Hajime Uno\",\"doi\":\"10.1002/sim.70056\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Given the limitations of using the Cox hazard ratio to summarize the magnitude of the treatment effect, alternative measures that do not have these limitations are gaining attention. One of the recently proposed alternative methods uses the average hazard with survival weight (AH). This population quantity can be interpreted as the average intensity of the event occurrence in a given time window that does not involve study-specific censoring. Inference procedures for the ratio of AH and difference in AH have already been proposed in simple randomized controlled trial settings to compare two groups. However, methods with stratification factors have not been well discussed, although stratified analysis is often used in practice to adjust for confounding factors and increase the power to detect a between-group difference. The conventional stratified analysis or meta-analysis approach, which integrates stratum-specific treatment effects using an optimal weight, directly applies to the ratio of AH and difference in AH. However, this conventional approach has significant limitations similar to the Cochran-Mantel-Haenszel method for a binary outcome and the stratified Cox procedure for a time-to-event outcome. To address this, we propose a new stratified analysis method for AH using standardization. With the proposed method, one can summarize the between-group treatment effect in both absolute difference and relative terms, adjusting for stratification factors. This can be a valuable alternative to the traditional stratified Cox procedure to estimate and report the magnitude of the treatment effect on time-to-event outcomes using hazard.</p>\",\"PeriodicalId\":21879,\"journal\":{\"name\":\"Statistics in Medicine\",\"volume\":\"44 7\",\"pages\":\"e70056\"},\"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.70056\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"MATHEMATICAL & COMPUTATIONAL BIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Statistics in Medicine","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/sim.70056","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"MATHEMATICAL & COMPUTATIONAL BIOLOGY","Score":null,"Total":0}
A Novel Stratified Analysis Method for Testing and Estimating Overall Treatment Effects on Time-To-Event Outcomes Using Average Hazard With Survival Weight.
Given the limitations of using the Cox hazard ratio to summarize the magnitude of the treatment effect, alternative measures that do not have these limitations are gaining attention. One of the recently proposed alternative methods uses the average hazard with survival weight (AH). This population quantity can be interpreted as the average intensity of the event occurrence in a given time window that does not involve study-specific censoring. Inference procedures for the ratio of AH and difference in AH have already been proposed in simple randomized controlled trial settings to compare two groups. However, methods with stratification factors have not been well discussed, although stratified analysis is often used in practice to adjust for confounding factors and increase the power to detect a between-group difference. The conventional stratified analysis or meta-analysis approach, which integrates stratum-specific treatment effects using an optimal weight, directly applies to the ratio of AH and difference in AH. However, this conventional approach has significant limitations similar to the Cochran-Mantel-Haenszel method for a binary outcome and the stratified Cox procedure for a time-to-event outcome. To address this, we propose a new stratified analysis method for AH using standardization. With the proposed method, one can summarize the between-group treatment effect in both absolute difference and relative terms, adjusting for stratification factors. This can be a valuable alternative to the traditional stratified Cox procedure to estimate and report the magnitude of the treatment effect on time-to-event outcomes using hazard.
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.