TY - JOUR
T1 - Idem
T2 - An R package for inferences in clinical trials with death and missingness
AU - Wang, Chenguang
AU - Colantuoni, Elizabeth
AU - Leroux, Andrew
AU - Scharfstein, Daniel O.
N1 - Funding Information:
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.
Publisher Copyright:
© 2020, American Statistical Association. All rights reserved.
PY - 2020
Y1 - 2020
N2 - 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.
AB - 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.
KW - Clinical trial
KW - Composite endpoint
KW - Imputation
KW - Missing data
KW - R
KW - SACE
KW - Sensitivity analysis
KW - Shiny
KW - Stan
KW - Truncation due to death
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U2 - 10.18637/jss.v093.i12
DO - 10.18637/jss.v093.i12
M3 - Article
C2 - 33273895
AN - SCOPUS:85085883420
VL - 93
SP - 1
EP - 35
JO - Journal of Statistical Software
JF - Journal of Statistical Software
SN - 1548-7660
IS - 12
ER -