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Understanding the natural progression in %FEV(1) decline in patients with cystic fibrosis: a longitudinal study

BACKGROUND: Forced expiratory volume in 1 s as a percentage of predicted (%FEV(1)) is a key outcome in cystic fibrosis (CF) and other lung diseases. As people with CF survive for longer periods, new methods are required to understand the way %FEV(1) changes over time. An up to date approach for long...

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Autores principales: Taylor-Robinson, David, Whitehead, Margaret, Diderichsen, Finn, Olesen, Hanne Vebert, Pressler, Tania, Smyth, Rosalind L, Diggle, Peter
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Group 2012
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3446776/
https://www.ncbi.nlm.nih.gov/pubmed/22555277
http://dx.doi.org/10.1136/thoraxjnl-2011-200953
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author Taylor-Robinson, David
Whitehead, Margaret
Diderichsen, Finn
Olesen, Hanne Vebert
Pressler, Tania
Smyth, Rosalind L
Diggle, Peter
author_facet Taylor-Robinson, David
Whitehead, Margaret
Diderichsen, Finn
Olesen, Hanne Vebert
Pressler, Tania
Smyth, Rosalind L
Diggle, Peter
author_sort Taylor-Robinson, David
collection PubMed
description BACKGROUND: Forced expiratory volume in 1 s as a percentage of predicted (%FEV(1)) is a key outcome in cystic fibrosis (CF) and other lung diseases. As people with CF survive for longer periods, new methods are required to understand the way %FEV(1) changes over time. An up to date approach for longitudinal modelling of %FEV(1) is presented and applied to a unique CF dataset to demonstrate its utility at the clinical and population level. METHODS AND FINDINGS: The Danish CF register contains 70 448 %FEV(1) measures on 479 patients seen monthly between 1969 and 2010. The variability in the data is partitioned into three components (between patient, within patient and measurement error) using the empirical variogram. Then a linear mixed effects model is developed to explore factors influencing %FEV(1) in this population. Lung function measures are correlated for over 15 years. A baseline %FEV(1) value explains 63% of the variability in %FEV(1) at 1 year, 40% at 3 years, and about 30% at 5 years. The model output smooths out the short-term variability in %FEV(1) (SD 6.3%), aiding clinical interpretation of changes in %FEV(1). At the population level significant effects of birth cohort, pancreatic status and Pseudomonas aeruginosa infection status on %FEV(1) are shown over time. CONCLUSIONS: This approach provides a more realistic estimate of the %FEV(1) trajectory of people with chronic lung disease by acknowledging the imprecision in individual measurements and the correlation structure of repeated measurements on the same individual over time. This method has applications for clinicians in assessing prognosis and the need for treatment intensification, and for use in clinical trials.
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spelling pubmed-34467762012-09-21 Understanding the natural progression in %FEV(1) decline in patients with cystic fibrosis: a longitudinal study Taylor-Robinson, David Whitehead, Margaret Diderichsen, Finn Olesen, Hanne Vebert Pressler, Tania Smyth, Rosalind L Diggle, Peter Thorax Original Articles BACKGROUND: Forced expiratory volume in 1 s as a percentage of predicted (%FEV(1)) is a key outcome in cystic fibrosis (CF) and other lung diseases. As people with CF survive for longer periods, new methods are required to understand the way %FEV(1) changes over time. An up to date approach for longitudinal modelling of %FEV(1) is presented and applied to a unique CF dataset to demonstrate its utility at the clinical and population level. METHODS AND FINDINGS: The Danish CF register contains 70 448 %FEV(1) measures on 479 patients seen monthly between 1969 and 2010. The variability in the data is partitioned into three components (between patient, within patient and measurement error) using the empirical variogram. Then a linear mixed effects model is developed to explore factors influencing %FEV(1) in this population. Lung function measures are correlated for over 15 years. A baseline %FEV(1) value explains 63% of the variability in %FEV(1) at 1 year, 40% at 3 years, and about 30% at 5 years. The model output smooths out the short-term variability in %FEV(1) (SD 6.3%), aiding clinical interpretation of changes in %FEV(1). At the population level significant effects of birth cohort, pancreatic status and Pseudomonas aeruginosa infection status on %FEV(1) are shown over time. CONCLUSIONS: This approach provides a more realistic estimate of the %FEV(1) trajectory of people with chronic lung disease by acknowledging the imprecision in individual measurements and the correlation structure of repeated measurements on the same individual over time. This method has applications for clinicians in assessing prognosis and the need for treatment intensification, and for use in clinical trials. BMJ Group 2012-10 2012-05-03 /pmc/articles/PMC3446776/ /pubmed/22555277 http://dx.doi.org/10.1136/thoraxjnl-2011-200953 Text en © 2012, Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions. This is an open-access article distributed under the terms of the Creative Commons Attribution Non-commercial License, which permits use, distribution, and reproduction in any medium, provided the original work is properly cited, the use is non commercial and is otherwise in compliance with the license. See: http://creativecommons.org/licenses/by-nc/2.0/ and http://creativecommons.org/licenses/by-nc/2.0/legalcode.
spellingShingle Original Articles
Taylor-Robinson, David
Whitehead, Margaret
Diderichsen, Finn
Olesen, Hanne Vebert
Pressler, Tania
Smyth, Rosalind L
Diggle, Peter
Understanding the natural progression in %FEV(1) decline in patients with cystic fibrosis: a longitudinal study
title Understanding the natural progression in %FEV(1) decline in patients with cystic fibrosis: a longitudinal study
title_full Understanding the natural progression in %FEV(1) decline in patients with cystic fibrosis: a longitudinal study
title_fullStr Understanding the natural progression in %FEV(1) decline in patients with cystic fibrosis: a longitudinal study
title_full_unstemmed Understanding the natural progression in %FEV(1) decline in patients with cystic fibrosis: a longitudinal study
title_short Understanding the natural progression in %FEV(1) decline in patients with cystic fibrosis: a longitudinal study
title_sort understanding the natural progression in %fev(1) decline in patients with cystic fibrosis: a longitudinal study
topic Original Articles
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3446776/
https://www.ncbi.nlm.nih.gov/pubmed/22555277
http://dx.doi.org/10.1136/thoraxjnl-2011-200953
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