{"title":"数据缺失情况下个性化治疗规则的鲁棒迁移学习。","authors":"Zhiyu Sui, Ying Ding, Lu Tang","doi":"10.1093/biostatistics/kxaf023","DOIUrl":null,"url":null,"abstract":"<p><p>Individualized treatment rule (ITR) is a stepping stone to precision medicine. To ensure validity, ITRs are ideally derived from randomized trial data, but the use cases of ITRs extend beyond these trial populations. Transferring knowledge from experimental data to real-world data is of interest, while experimental data with selective inclusion criteria reflect a population distribution that may differ from the real-world target. In well-designed experiments, granular information crucial to decision making can be thoroughly collected. However, part of this may not be accessible in real-world scenarios. We propose a learning scheme for ITR that simultaneously addresses the issues of covariate shift and missing covariates with a quantile-based optimal treatment objective. Specifically, we compare the outcome uncertainty across treatment arms that is due to missing covariates and use it to guide treatment selection to reduce the likelihood of worse outcomes. The performance of this method is evaluated in simulations and a sepsis data application.</p>","PeriodicalId":55357,"journal":{"name":"Biostatistics","volume":"26 1","pages":""},"PeriodicalIF":2.0000,"publicationDate":"2024-12-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12342780/pdf/","citationCount":"0","resultStr":"{\"title\":\"Robust transfer learning for individualized treatment rules in the presence of missing data.\",\"authors\":\"Zhiyu Sui, Ying Ding, Lu Tang\",\"doi\":\"10.1093/biostatistics/kxaf023\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Individualized treatment rule (ITR) is a stepping stone to precision medicine. To ensure validity, ITRs are ideally derived from randomized trial data, but the use cases of ITRs extend beyond these trial populations. Transferring knowledge from experimental data to real-world data is of interest, while experimental data with selective inclusion criteria reflect a population distribution that may differ from the real-world target. In well-designed experiments, granular information crucial to decision making can be thoroughly collected. However, part of this may not be accessible in real-world scenarios. We propose a learning scheme for ITR that simultaneously addresses the issues of covariate shift and missing covariates with a quantile-based optimal treatment objective. Specifically, we compare the outcome uncertainty across treatment arms that is due to missing covariates and use it to guide treatment selection to reduce the likelihood of worse outcomes. The performance of this method is evaluated in simulations and a sepsis data application.</p>\",\"PeriodicalId\":55357,\"journal\":{\"name\":\"Biostatistics\",\"volume\":\"26 1\",\"pages\":\"\"},\"PeriodicalIF\":2.0000,\"publicationDate\":\"2024-12-31\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12342780/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Biostatistics\",\"FirstCategoryId\":\"100\",\"ListUrlMain\":\"https://doi.org/10.1093/biostatistics/kxaf023\",\"RegionNum\":3,\"RegionCategory\":\"数学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"MATHEMATICAL & COMPUTATIONAL BIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Biostatistics","FirstCategoryId":"100","ListUrlMain":"https://doi.org/10.1093/biostatistics/kxaf023","RegionNum":3,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"MATHEMATICAL & COMPUTATIONAL BIOLOGY","Score":null,"Total":0}
Robust transfer learning for individualized treatment rules in the presence of missing data.
Individualized treatment rule (ITR) is a stepping stone to precision medicine. To ensure validity, ITRs are ideally derived from randomized trial data, but the use cases of ITRs extend beyond these trial populations. Transferring knowledge from experimental data to real-world data is of interest, while experimental data with selective inclusion criteria reflect a population distribution that may differ from the real-world target. In well-designed experiments, granular information crucial to decision making can be thoroughly collected. However, part of this may not be accessible in real-world scenarios. We propose a learning scheme for ITR that simultaneously addresses the issues of covariate shift and missing covariates with a quantile-based optimal treatment objective. Specifically, we compare the outcome uncertainty across treatment arms that is due to missing covariates and use it to guide treatment selection to reduce the likelihood of worse outcomes. The performance of this method is evaluated in simulations and a sepsis data application.
期刊介绍:
Among the important scientific developments of the 20th century is the explosive growth in statistical reasoning and methods for application to studies of human health. Examples include developments in likelihood methods for inference, epidemiologic statistics, clinical trials, survival analysis, and statistical genetics. Substantive problems in public health and biomedical research have fueled the development of statistical methods, which in turn have improved our ability to draw valid inferences from data. The objective of Biostatistics is to advance statistical science and its application to problems of human health and disease, with the ultimate goal of advancing the public''s health.