Nonparametric Benefit-Risk Assessment Using Marker Process in the Presence of a Terminal Event

2017
Benefitrisk assessment is a crucial step in medical decision process. In many biomedical studies, both longitudinal marker measurements and time to a terminal event serve as important endpoints for benefitrisk assessment. The effect of an intervention or a treatment on the longitudinal marker process, however, can be in conflict with its effect on the time to the terminal event. Thus, questions arise on how to evaluate treatment effects based on the two endpoints, for the purpose of deciding on which treatment is most likely to benefit the patients. In this article, we present a unified framework for benefitrisk assessment using the observed longitudinal markers and time to event data. We propose a cumulative weighted marker process to synthesize information from the two endpoints, and use its mean function at a prespecified time point as a benefitrisk summary measure. We consider nonparametric estimation of the summary measure under two scenarios: (i) the longitudinal marker is measured intermittently during the study period, and (ii) the value of the longitudinal marker is observed throughout the entire follow-up period. The large-sample properties of the estimators are derived and compared. Simulation studies and data examples exhibit that the proposed methods are easy to implement and reliable for practical use. Supplemental materials for this article are available online.
JOURNAL OF THE AMERICAN STATISTICAL ASSOCIATION
页码:826-836|卷号:112|期号:518
ISSN:0162-1459
收录类型
SSCI
发表日期
2017
学科领域
循证社会科学-方法
国家
美国
语种
英语
DOI
10.1080/01621459.2016.1180988
其他关键词
QUALITY-ADJUSTED SURVIVAL; COST-EFFECTIVENESS ANALYSIS; LONGITUDINAL DATA; TIME; ESTIMATORS; HEALTH; SAMPLE; TRIAL; AIDS
EISSN
1537-274X
资助机构
National Institute of HealthUnited States Department of Health & Human ServicesNational Institutes of Health (NIH) - USA [R01HL122212, R01CA193888]; NATIONAL CANCER INSTITUTEUnited States Department of Health & Human ServicesNational Institutes of Health (NIH) - USANIH National Cancer Institute (NCI) [R01CA193888] Funding Source: NIH RePORTER; NATIONAL HEART, LUNG, AND BLOOD INSTITUTEUnited States Department of Health & Human ServicesNational Institutes of Health (NIH) - USANIH National Heart Lung & Blood Institute (NHLBI) [R01HL122212] Funding Source: NIH RePORTER; NATIONAL INSTITUTE ON AGINGUnited States Department of Health & Human ServicesNational Institutes of Health (NIH) - USANIH National Institute on Aging (NIA) [P50AG005146] Funding Source: NIH RePORTER
资助信息
The research was supported by the National Institute of Health grants R01HL122212 and R01CA193888.
被引频次(WOS)
2
被引更新日期
2022-01
来源机构
Johns Hopkins University Johns Hopkins Bloomberg School of Public Health Johns Hopkins University Johns Hopkins Medicine
关键词
Kernel smoothing Longitudinal marker process Multiple event process Survival analysis