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Evolution of lung function during the first year of life in newborn screened cystic fibrosis infants

RATIONALE: Newborn screening (NBS) for cystic fibrosis (CF) allows early intervention. Design of randomised controlled trials (RCT) is currently impeded by uncertainty regarding evolution of lung function, an important trial end point in such infants. OBJECTIVE: To assess changes in pulmonary functi...

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Autores principales: Nguyen, The Thanh-Diem, Thia, Lena P, Hoo, Ah-Fong, Bush, Andrew, Aurora, Paul, Wade, Angie, Chudleigh, Jane, Lum, Sooky, Stocks, Janet
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4174068/
https://www.ncbi.nlm.nih.gov/pubmed/24072358
http://dx.doi.org/10.1136/thoraxjnl-2013-204023
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author Nguyen, The Thanh-Diem
Thia, Lena P
Hoo, Ah-Fong
Bush, Andrew
Aurora, Paul
Wade, Angie
Chudleigh, Jane
Lum, Sooky
Stocks, Janet
author_facet Nguyen, The Thanh-Diem
Thia, Lena P
Hoo, Ah-Fong
Bush, Andrew
Aurora, Paul
Wade, Angie
Chudleigh, Jane
Lum, Sooky
Stocks, Janet
author_sort Nguyen, The Thanh-Diem
collection PubMed
description RATIONALE: Newborn screening (NBS) for cystic fibrosis (CF) allows early intervention. Design of randomised controlled trials (RCT) is currently impeded by uncertainty regarding evolution of lung function, an important trial end point in such infants. OBJECTIVE: To assess changes in pulmonary function during the first year of life in CF NBS infants. METHODS: Observational longitudinal study. CF NBS infants and healthy controls were recruited between 2009 and 2011. Lung Clearance Index (LCI), plethysmographic lung volume (plethysmographic functional residual capacity (FRC(pleth))) and forced expired volume (FEV(0.5)) were measured at 3 months and 1 year of age. MAIN RESULTS: Paired measurements were obtained from 72 CF infants and 44 controls. At 3 months, CF infants had significantly worse lung function for all tests. FEV(0.5) improved significantly (0.59 (95% CI 0.18 to 0.99) z-scores; p<0.01) in CF infants between 3 months and 1 year, and by 1 year, FEV(0.5) was only 0.52 (0.89 to 0.15) z-scores less than in controls. LCI and FRC(pleth) remained stable throughout the first year of life, being on average 0.8 z-scores higher in infants with CF. Pulmonary function at 1 year was predicted by that at 3 months. Among the 45 CF infants with entirely normal LCI and FEV(0.5) at 3 months, 80% remained so at 1 year, while 74% of those with early abnormalities remained abnormal at 1 year. CONCLUSIONS: This is the first study reporting improvements in FEV(0.5) over time in stable NBS CF infants treated with standard therapy. Milder changes in lung function occurred by 1 year than previously reported. Lung function at 3 months predicts a high-risk group, who should be considered for intensification of treatment and enrolment into RCTs.
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spelling pubmed-41740682014-10-02 Evolution of lung function during the first year of life in newborn screened cystic fibrosis infants Nguyen, The Thanh-Diem Thia, Lena P Hoo, Ah-Fong Bush, Andrew Aurora, Paul Wade, Angie Chudleigh, Jane Lum, Sooky Stocks, Janet Thorax Cystic Fibrosis RATIONALE: Newborn screening (NBS) for cystic fibrosis (CF) allows early intervention. Design of randomised controlled trials (RCT) is currently impeded by uncertainty regarding evolution of lung function, an important trial end point in such infants. OBJECTIVE: To assess changes in pulmonary function during the first year of life in CF NBS infants. METHODS: Observational longitudinal study. CF NBS infants and healthy controls were recruited between 2009 and 2011. Lung Clearance Index (LCI), plethysmographic lung volume (plethysmographic functional residual capacity (FRC(pleth))) and forced expired volume (FEV(0.5)) were measured at 3 months and 1 year of age. MAIN RESULTS: Paired measurements were obtained from 72 CF infants and 44 controls. At 3 months, CF infants had significantly worse lung function for all tests. FEV(0.5) improved significantly (0.59 (95% CI 0.18 to 0.99) z-scores; p<0.01) in CF infants between 3 months and 1 year, and by 1 year, FEV(0.5) was only 0.52 (0.89 to 0.15) z-scores less than in controls. LCI and FRC(pleth) remained stable throughout the first year of life, being on average 0.8 z-scores higher in infants with CF. Pulmonary function at 1 year was predicted by that at 3 months. Among the 45 CF infants with entirely normal LCI and FEV(0.5) at 3 months, 80% remained so at 1 year, while 74% of those with early abnormalities remained abnormal at 1 year. CONCLUSIONS: This is the first study reporting improvements in FEV(0.5) over time in stable NBS CF infants treated with standard therapy. Milder changes in lung function occurred by 1 year than previously reported. Lung function at 3 months predicts a high-risk group, who should be considered for intensification of treatment and enrolment into RCTs. BMJ Publishing Group 2014-10 2013-09-26 /pmc/articles/PMC4174068/ /pubmed/24072358 http://dx.doi.org/10.1136/thoraxjnl-2013-204023 Text en 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 in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 3.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/3.0/
spellingShingle Cystic Fibrosis
Nguyen, The Thanh-Diem
Thia, Lena P
Hoo, Ah-Fong
Bush, Andrew
Aurora, Paul
Wade, Angie
Chudleigh, Jane
Lum, Sooky
Stocks, Janet
Evolution of lung function during the first year of life in newborn screened cystic fibrosis infants
title Evolution of lung function during the first year of life in newborn screened cystic fibrosis infants
title_full Evolution of lung function during the first year of life in newborn screened cystic fibrosis infants
title_fullStr Evolution of lung function during the first year of life in newborn screened cystic fibrosis infants
title_full_unstemmed Evolution of lung function during the first year of life in newborn screened cystic fibrosis infants
title_short Evolution of lung function during the first year of life in newborn screened cystic fibrosis infants
title_sort evolution of lung function during the first year of life in newborn screened cystic fibrosis infants
topic Cystic Fibrosis
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4174068/
https://www.ncbi.nlm.nih.gov/pubmed/24072358
http://dx.doi.org/10.1136/thoraxjnl-2013-204023
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