Event-specific win ratios for inference with terminal and non-terminal events.
multiple endpoints
proportional hazards
semi-competing risks data
survival analysis
treatment effect
Journal
Statistics in medicine
ISSN: 1097-0258
Titre abrégé: Stat Med
Pays: England
ID NLM: 8215016
Informations de publication
Date de publication:
30 03 2022
30 03 2022
Historique:
revised:
15
10
2021
received:
29
04
2021
accepted:
03
11
2021
pubmed:
25
11
2021
medline:
6
4
2022
entrez:
24
11
2021
Statut:
ppublish
Résumé
For semi-competing risks data involving a non-terminal event and a terminal event we derive the asymptotic distributions of the event-specific win ratios under proportional hazards (PH) assumptions for the relevant cause-specific hazard functions of the non-terminal and terminal event, respectively. The win ratios converge to the respective hazard ratios under the PH assumptions and therefore are censoring-free, whether or not the censoring distributions in the two treatment arms are the same. With the asymptotic bivariate normal distributions of the win ratios, confidence intervals and testing procedures are obtained. Through extensive simulation studies and data analysis, we identified proper transformations of the win ratios that yield good control of the type one error rate for various testing procedures while maintaining competitive power. The confidence intervals also have good coverage probabilities. Furthermore, a test for the PH assumptions and a test of equal hazard ratios are developed. The new procedures are illustrated in the clinical trial Aldosterone Antagonist Therapy for Adults With Heart Failure and Preserved Systolic Function, which evaluated the effects of spironolactone in patients with heart failure and a preserved left ventricular ejection fraction.
Substances chimiques
Mineralocorticoid Receptor Antagonists
0
Spironolactone
27O7W4T232
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
1225-1241Informations de copyright
© 2021 John Wiley & Sons Ltd. This article has been contributed to by US Government employees and their work is in the public domain in the USA.
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