Bivariate pseudo-observations for recurrent event analysis with terminal events

Publikation: Bidrag til tidsskriftTidsskriftartikelForskningfagfællebedømt

The analysis of recurrent events in the presence of terminal events requires special attention. Several approaches have been suggested for such analyses either using intensity models or marginal models. When analysing treatment effects on recurrent events in controlled trials, special attention should be paid to competing deaths and their impact on interpretation. This paper proposes a method that formulates a marginal model for recurrent events and terminal events simultaneously. Estimation is based on pseudo-observations for both the expected number of events and survival probabilities. Various relevant hypothesis tests in the framework are explored. Theoretical derivations and simulation studies are conducted to investigate the behaviour of the method. The method is applied to two real data examples. The bivariate marginal pseudo-observation model carries the strength of a two-dimensional modelling procedure and performs well in comparison with available models. Finally, an extension to a three-dimensional model, which decomposes the terminal event per death cause, is proposed and exemplified.

OriginalsprogEngelsk
TidsskriftLifetime Data Analysis
Vol/bind29
Sider (fra-til)256-287
Antal sider12
ISSN1380-7870
DOI
StatusUdgivet - 2023

Bibliografisk note

Funding Information:
This research was carried out as part of xxx’s Ph.D. education. For the PhD education, she received funding from Novo Nordisk A/S and Innovation Fund Denmark. xxx is supported by the Novo Nordisk Foundation Grant NNF17OC0028276.

Publisher Copyright:
© 2021, The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.

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