idem: An R Package for Inferences in Clinical Trials with Death and Missingness

2020
In randomized controlled trials of seriously ill patients, death is common and often defined as the primary endpoint. Increasingly, non-mortality outcomes such as functional outcomes are co-primary or secondary endpoints. Functional outcomes are not defined for patients who die, referred to as truncation due to death, and among survivors, functional outcomes are often unobserved due to missed clinic visits or loss to follow-up. It is well known that if the functional outcomes truncated due to death or missing are handled inappropriately, treatment effect estimation can be biased. In this paper, we describe the package idem that implements a procedure for comparing treatments that is based on a composite endpoint of mortality and the functional outcome among survivors. Among survivors, the procedure incorporates a missing data imputation procedure with a sensitivity analysis strategy. A web-based graphical user interface is provided in the idem package to facilitate users conducting the proposed analysis in an interactive and user-friendly manner. We demonstrate idem using data from a recent trial of sedation interruption among mechanically ventilated patients.
JOURNAL OF STATISTICAL SOFTWARE
页码:1-35|卷号:93|期号:12
ISSN:1548-7660
收录类型
SSCI
发表日期
2020
学科领域
循证社会科学-方法
国家
美国
语种
英语
DOI
10.18637/jss.v093.i12
资助机构
FDA, NIH [R24HL111895]; PCORI, NIHUnited States Department of Health & Human ServicesNational Institutes of Health (NIH) - USA [R24HL111895]; NCIUnited States Department of Health & Human ServicesNational Institutes of Health (NIH) - USANIH National Cancer Institute (NCI) [CA183854]; Cervical Cancer SPORE [NCI 1 P50 CA098252]; National Science FoundationNational Science Foundation (NSF) [ACI-1548562]
资助信息
The work was partially supported by contracts from FDA and PCORI, NIH grant number R24HL111895, NCI grant number CA183854 and the Cervical Cancer SPORE (NCI 1 P50 CA098252). This work used the Extreme Science and Engineering Discovery Environment (XSEDE; Towns et al. 2014), which is supported by National Science Foundation grant number ACI-1548562. We are also immensely grateful to the two anonymous reviewers for their insightful comments on an earlier version of the manuscript.
被引频次(WOS)
0
被引更新日期
2022-01
来源机构
Johns Hopkins University Johns Hopkins Medicine Johns Hopkins University Johns Hopkins Bloomberg School of Public Health
关键词
clinical trial truncation due to death composite endpoint imputation missing data R SACE sensitivity analysis shiny Stan