Direct modeling of the crude probability of cancer death and the number of life years lost due to cancer without the need of cause of death: a pseudo-observation approach in the relative survival setting.

Dimitra-Kleio Kipourou ORCID logo; Maja Pohar Perme; Bernard Rachet ORCID logo; Aurelien Belot ORCID logo; (2020) Direct modeling of the crude probability of cancer death and the number of life years lost due to cancer without the need of cause of death: a pseudo-observation approach in the relative survival setting. Biostatistics, 23 (1). pp. 101-119. DOI: 10.1093/biostatistics/kxaa017
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In population-based cancer studies, net survival is a crucial measure for population comparison purposes. However, alternative measures, namely the crude probability of death (CPr) and the number of life years lost (LYL) due to death according to different causes, are useful as complementary measures for reflecting different dimensions in terms of prognosis, treatment choice, or development of a control strategy. When the cause of death (COD) information is available, both measures can be estimated in competing risks setting using either cause-specific or subdistribution hazard regression models or with the pseudo-observation approach through direct modeling. We extended the pseudo-observation approach in order to model the CPr and the LYL due to different causes when information on COD is unavailable or unreliable (i.e., in relative survival setting). In a simulation study, we assessed the performance of the proposed approach in estimating regression parameters and examined models with different link functions that can provide an easier interpretation of the parameters. We showed that the pseudo-observation approach performs well for both measures and we illustrated their use on cervical cancer data from the England population-based cancer registry. A tutorial showing how to implement the method in R software is also provided.


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