The survival-incorporated median vs the median in the survivors or in the always-survivors: What are we measuring? and Why?
estimands
survival
survival-incorporated median
survivor average causal effect
treatment effect
truncation by death
Journal
Statistics in medicine
ISSN: 1097-0258
Titre abrégé: Stat Med
Pays: England
ID NLM: 8215016
Informations de publication
Date de publication:
20 12 2023
20 12 2023
Historique:
received:
26
04
2023
accepted:
13
09
2023
medline:
20
11
2023
pubmed:
13
10
2023
entrez:
12
10
2023
Statut:
ppublish
Résumé
Many clinical studies evaluate the benefit of a treatment based on both survival and other continuous/ordinal clinical outcomes, such as quality of life scores. In these studies, when subjects die before the follow-up assessment, the clinical outcomes become undefined and are truncated by death. Treating outcomes as "missing" or "censored" due to death can be misleading for treatment effect evaluation. We show that if we use the median in the survivors or in the always-survivors as estimands to summarize clinical outcomes, we may conclude that a trade-off exists between the probability of survival and good clinical outcomes, even in settings where both the probability of survival and the probability of any good clinical outcome are better for one treatment. Therefore, we advocate not always treating death as a mechanism through which clinical outcomes are missing, but rather as part of the outcome measure. To account for the survival status, we describe the survival-incorporated median as an alternative summary measure for outcomes in the presence of death. The survival-incorporated median is the threshold such that 50% of the population is alive with an outcome above that threshold. Through conceptual examples and an application to a prostate cancer treatment study, we show that the survival-incorporated median provides a simple and useful summary measure to inform clinical practice.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
5479-5490Subventions
Organisme : NIAID NIH HHS
ID : UM1 AI068634
Pays : United States
Informations de copyright
© 2023 John Wiley & Sons Ltd.
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