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Estimating restricted mean survival time and expected life-years lost in the presence of competing risks within flexible parametric survival models
BACKGROUND: Royston-Parmar flexible parametric survival models (FPMs) can be fitted on either the cause-specific hazards or cumulative incidence scale in the presence of competing risks. An advantage of modelling within this framework for competing risks data is the ease at which alternative predict...
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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BioMed Central
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7953595/ https://www.ncbi.nlm.nih.gov/pubmed/33706711 http://dx.doi.org/10.1186/s12874-021-01213-0 |
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author | Mozumder, Sarwar I. Rutherford, Mark J. Lambert, Paul C. |
author_facet | Mozumder, Sarwar I. Rutherford, Mark J. Lambert, Paul C. |
author_sort | Mozumder, Sarwar I. |
collection | PubMed |
description | BACKGROUND: Royston-Parmar flexible parametric survival models (FPMs) can be fitted on either the cause-specific hazards or cumulative incidence scale in the presence of competing risks. An advantage of modelling within this framework for competing risks data is the ease at which alternative predictions to the (cause-specific or subdistribution) hazard ratio can be obtained. Restricted mean survival time (RMST), or restricted mean failure time (RMFT) on the mortality scale, is one such measure. This has an attractive interpretation, especially when the proportionality assumption is violated. Compared to similar measures, fewer assumptions are required and it does not require extrapolation. Furthermore, one can easily obtain the expected number of life-years lost, or gained, due to a particular cause of death, which is a further useful prognostic measure as introduced by Andersen. METHODS: In the presence of competing risks, prediction of RMFT and the expected life-years lost due to a cause of death are presented using Royston-Parmar FPMs. These can be predicted for a specific covariate pattern to facilitate interpretation in observational studies at the individual level, or at the population-level using standardisation to obtain marginal measures. Predictions are illustrated using English colorectal data and are obtained using the Stata post-estimation command, standsurv. RESULTS: Reporting such measures facilitate interpretation of a competing risks analysis, particularly when the proportional hazards assumption is not appropriate. Standardisation provides a useful way to obtain marginal estimates to make absolute comparisons between two covariate groups. Predictions can be made at various time-points and presented visually for each cause of death to better understand the overall impact of different covariate groups. CONCLUSIONS: We describe estimation of RMFT, and expected life-years lost partitioned by each competing cause of death after fitting a single FPM on either the log-cumulative subdistribution, or cause-specific hazards scale. These can be used to facilitate interpretation of a competing risks analysis when the proportionality assumption is in doubt. |
format | Online Article Text |
id | pubmed-7953595 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
spelling | pubmed-79535952021-03-12 Estimating restricted mean survival time and expected life-years lost in the presence of competing risks within flexible parametric survival models Mozumder, Sarwar I. Rutherford, Mark J. Lambert, Paul C. BMC Med Res Methodol Research Article BACKGROUND: Royston-Parmar flexible parametric survival models (FPMs) can be fitted on either the cause-specific hazards or cumulative incidence scale in the presence of competing risks. An advantage of modelling within this framework for competing risks data is the ease at which alternative predictions to the (cause-specific or subdistribution) hazard ratio can be obtained. Restricted mean survival time (RMST), or restricted mean failure time (RMFT) on the mortality scale, is one such measure. This has an attractive interpretation, especially when the proportionality assumption is violated. Compared to similar measures, fewer assumptions are required and it does not require extrapolation. Furthermore, one can easily obtain the expected number of life-years lost, or gained, due to a particular cause of death, which is a further useful prognostic measure as introduced by Andersen. METHODS: In the presence of competing risks, prediction of RMFT and the expected life-years lost due to a cause of death are presented using Royston-Parmar FPMs. These can be predicted for a specific covariate pattern to facilitate interpretation in observational studies at the individual level, or at the population-level using standardisation to obtain marginal measures. Predictions are illustrated using English colorectal data and are obtained using the Stata post-estimation command, standsurv. RESULTS: Reporting such measures facilitate interpretation of a competing risks analysis, particularly when the proportional hazards assumption is not appropriate. Standardisation provides a useful way to obtain marginal estimates to make absolute comparisons between two covariate groups. Predictions can be made at various time-points and presented visually for each cause of death to better understand the overall impact of different covariate groups. CONCLUSIONS: We describe estimation of RMFT, and expected life-years lost partitioned by each competing cause of death after fitting a single FPM on either the log-cumulative subdistribution, or cause-specific hazards scale. These can be used to facilitate interpretation of a competing risks analysis when the proportionality assumption is in doubt. BioMed Central 2021-03-11 /pmc/articles/PMC7953595/ /pubmed/33706711 http://dx.doi.org/10.1186/s12874-021-01213-0 Text en © The Author(s) 2021 Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data. |
spellingShingle | Research Article Mozumder, Sarwar I. Rutherford, Mark J. Lambert, Paul C. Estimating restricted mean survival time and expected life-years lost in the presence of competing risks within flexible parametric survival models |
title | Estimating restricted mean survival time and expected life-years lost in the presence of competing risks within flexible parametric survival models |
title_full | Estimating restricted mean survival time and expected life-years lost in the presence of competing risks within flexible parametric survival models |
title_fullStr | Estimating restricted mean survival time and expected life-years lost in the presence of competing risks within flexible parametric survival models |
title_full_unstemmed | Estimating restricted mean survival time and expected life-years lost in the presence of competing risks within flexible parametric survival models |
title_short | Estimating restricted mean survival time and expected life-years lost in the presence of competing risks within flexible parametric survival models |
title_sort | estimating restricted mean survival time and expected life-years lost in the presence of competing risks within flexible parametric survival models |
topic | Research Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7953595/ https://www.ncbi.nlm.nih.gov/pubmed/33706711 http://dx.doi.org/10.1186/s12874-021-01213-0 |
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