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Registry-based study in people with cystic fibrosis and an R117H variant treated with ivacaftor

BACKGROUND: Ivacaftor approval was extended to people with cystic fibrosis (CF) and an R117H variant in 2014 in the USA. This observational, real-world, postapproval study evaluated long-term outcomes among people with CF and an R117H variant on ivacaftor using data from the US Cystic Fibrosis Found...

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Autores principales: Higgins, Mark, Farietta, Thalia, Campbell, Daniel, Liu, Meng, Ostrenga, Josh, Elbert, Alexander, Shih, Judy, Volkova, Nataliya
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10231020/
https://www.ncbi.nlm.nih.gov/pubmed/37230763
http://dx.doi.org/10.1136/bmjresp-2022-001447
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author Higgins, Mark
Farietta, Thalia
Campbell, Daniel
Liu, Meng
Ostrenga, Josh
Elbert, Alexander
Shih, Judy
Volkova, Nataliya
author_facet Higgins, Mark
Farietta, Thalia
Campbell, Daniel
Liu, Meng
Ostrenga, Josh
Elbert, Alexander
Shih, Judy
Volkova, Nataliya
author_sort Higgins, Mark
collection PubMed
description BACKGROUND: Ivacaftor approval was extended to people with cystic fibrosis (CF) and an R117H variant in 2014 in the USA. This observational, real-world, postapproval study evaluated long-term outcomes among people with CF and an R117H variant on ivacaftor using data from the US Cystic Fibrosis Foundation Patient Registry. METHODS: Key outcomes were evaluated in ivacaftor-treated people with CF and an R117H variant for up to 36 months before and after treatment initiation using within-group comparisons. Analyses were descriptive in nature, focused on evaluation of observed outcome patterns over time and were performed both overall and for age groups ≥2 to <6 years, ≥6 to <18 years and ≥18 years. Key outcomes included lung function, body mass index (BMI), pulmonary exacerbations (PEx) and hospitalisations. RESULTS: The ivacaftor cohort included 369 people with CF and an R117H variant who initiated therapy between 1 January 2015 and 31 December 2016. During each of the 12-month intervals following treatment initiation, the mean observed percent predicted forced expiratory volume in 1 s (ppFEV(1)) and BMI values were higher and the mean annualised number of PEx and hospitalisation events were lower than pretreatment values. Mean change in ppFEV(1) from pretreatment baseline was an increase of 1.5 (95% CI 0.8 to 2.3), 1.7 (95% CI 0.7 to 2.7) and 1.8 (95% CI 0.6 to 3.0) percentage points in the first, second and third years of treatment, respectively. Similar trends were observed in adult and paediatric subgroups. CONCLUSIONS: The results support the clinical effectiveness of ivacaftor in people with CF and an R117H variant, including adult and paediatric subgroups.
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spelling pubmed-102310202023-06-01 Registry-based study in people with cystic fibrosis and an R117H variant treated with ivacaftor Higgins, Mark Farietta, Thalia Campbell, Daniel Liu, Meng Ostrenga, Josh Elbert, Alexander Shih, Judy Volkova, Nataliya BMJ Open Respir Res Cystic Fibrosis BACKGROUND: Ivacaftor approval was extended to people with cystic fibrosis (CF) and an R117H variant in 2014 in the USA. This observational, real-world, postapproval study evaluated long-term outcomes among people with CF and an R117H variant on ivacaftor using data from the US Cystic Fibrosis Foundation Patient Registry. METHODS: Key outcomes were evaluated in ivacaftor-treated people with CF and an R117H variant for up to 36 months before and after treatment initiation using within-group comparisons. Analyses were descriptive in nature, focused on evaluation of observed outcome patterns over time and were performed both overall and for age groups ≥2 to <6 years, ≥6 to <18 years and ≥18 years. Key outcomes included lung function, body mass index (BMI), pulmonary exacerbations (PEx) and hospitalisations. RESULTS: The ivacaftor cohort included 369 people with CF and an R117H variant who initiated therapy between 1 January 2015 and 31 December 2016. During each of the 12-month intervals following treatment initiation, the mean observed percent predicted forced expiratory volume in 1 s (ppFEV(1)) and BMI values were higher and the mean annualised number of PEx and hospitalisation events were lower than pretreatment values. Mean change in ppFEV(1) from pretreatment baseline was an increase of 1.5 (95% CI 0.8 to 2.3), 1.7 (95% CI 0.7 to 2.7) and 1.8 (95% CI 0.6 to 3.0) percentage points in the first, second and third years of treatment, respectively. Similar trends were observed in adult and paediatric subgroups. CONCLUSIONS: The results support the clinical effectiveness of ivacaftor in people with CF and an R117H variant, including adult and paediatric subgroups. BMJ Publishing Group 2023-05-25 /pmc/articles/PMC10231020/ /pubmed/37230763 http://dx.doi.org/10.1136/bmjresp-2022-001447 Text en © Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. https://creativecommons.org/licenses/by-nc/4.0/This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) 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, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) .
spellingShingle Cystic Fibrosis
Higgins, Mark
Farietta, Thalia
Campbell, Daniel
Liu, Meng
Ostrenga, Josh
Elbert, Alexander
Shih, Judy
Volkova, Nataliya
Registry-based study in people with cystic fibrosis and an R117H variant treated with ivacaftor
title Registry-based study in people with cystic fibrosis and an R117H variant treated with ivacaftor
title_full Registry-based study in people with cystic fibrosis and an R117H variant treated with ivacaftor
title_fullStr Registry-based study in people with cystic fibrosis and an R117H variant treated with ivacaftor
title_full_unstemmed Registry-based study in people with cystic fibrosis and an R117H variant treated with ivacaftor
title_short Registry-based study in people with cystic fibrosis and an R117H variant treated with ivacaftor
title_sort registry-based study in people with cystic fibrosis and an r117h variant treated with ivacaftor
topic Cystic Fibrosis
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10231020/
https://www.ncbi.nlm.nih.gov/pubmed/37230763
http://dx.doi.org/10.1136/bmjresp-2022-001447
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