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Originalsprache | Englisch |
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Publikationsstatus | Elektronisch veröffentlicht (E-Pub) - 22 Juli 2020 |
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2020.
Publikation: Arbeitspapier/Preprint › Preprint
}
TY - UNPB
T1 - Model-based simultaneous inference for multiple subgroups and multiple endpoints
AU - Vogel, Charlotte
AU - Schaarschmidt, Frank
AU - Ritz, Christian
AU - Koenig, Franz
AU - Hothorn, Ludwig A.
PY - 2020/7/22
Y1 - 2020/7/22
N2 - Various methodological options exist on evaluating differences in both subgroups and the overall population. Most desirable is the simultaneous study of multiple endpoints in several populations. We investigate a newer method using multiple marginal models (mmm) which allows flexible handling of multiple endpoints, including continuous, binary or time-to-event data. This paper explores the performance of mmm in contrast to the standard Bonferroni approach via simulation. Mainly these methods are compared on the basis of their familywise error rate and power under different scenarios, varying in sample size and standard deviation. Additionally, it is shown that the method can deal with overlapping subgroup definitions and different combinations of endpoints may be assumed. The reanalysis of a clinical example shows a practical application.
AB - Various methodological options exist on evaluating differences in both subgroups and the overall population. Most desirable is the simultaneous study of multiple endpoints in several populations. We investigate a newer method using multiple marginal models (mmm) which allows flexible handling of multiple endpoints, including continuous, binary or time-to-event data. This paper explores the performance of mmm in contrast to the standard Bonferroni approach via simulation. Mainly these methods are compared on the basis of their familywise error rate and power under different scenarios, varying in sample size and standard deviation. Additionally, it is shown that the method can deal with overlapping subgroup definitions and different combinations of endpoints may be assumed. The reanalysis of a clinical example shows a practical application.
KW - stat.AP
M3 - Preprint
BT - Model-based simultaneous inference for multiple subgroups and multiple endpoints
ER -