CFTR modulator therapies – Effect on life expectancy in people with cystic fibrosis

CFTR modulators have dramatically changed the clinical course of CF in those fortunate enough to receive them. Inevitably, randomised controlled trials during the development of these drugs are too short to use mortality as an outcome. Evidence for their effect on life expectancy are best gained fro...

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Detalles Bibliográficos
Autores principales: Balfour-Lynn, I.M., King, J.A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier Ltd. 2022
Materias:
Mini-symposium: CF in the age of modulators (Part I)
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7255286/
https://www.ncbi.nlm.nih.gov/pubmed/32565113
http://dx.doi.org/10.1016/j.prrv.2020.05.002
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author Balfour-Lynn, I.M.
King, J.A.
author_facet Balfour-Lynn, I.M.
King, J.A.
author_sort Balfour-Lynn, I.M.
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description CFTR modulators have dramatically changed the clinical course of CF in those fortunate enough to receive them. Inevitably, randomised controlled trials during the development of these drugs are too short to use mortality as an outcome. Evidence for their effect on life expectancy are best gained from real world registry studies specifically looking at mortality, but these are only available for ivacaftor to date. Therefore, indirect evidence must be obtained by looking at outcomes known to affect mortality and seeing the effect of these drugs on those outcomes.
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spelling pubmed-72552862020-05-28 CFTR modulator therapies – Effect on life expectancy in people with cystic fibrosis Balfour-Lynn, I.M. King, J.A. Paediatr Respir Rev Mini-symposium: CF in the age of modulators (Part I) CFTR modulators have dramatically changed the clinical course of CF in those fortunate enough to receive them. Inevitably, randomised controlled trials during the development of these drugs are too short to use mortality as an outcome. Evidence for their effect on life expectancy are best gained from real world registry studies specifically looking at mortality, but these are only available for ivacaftor to date. Therefore, indirect evidence must be obtained by looking at outcomes known to affect mortality and seeing the effect of these drugs on those outcomes. Elsevier Ltd. 2022-06 2020-05-26 /pmc/articles/PMC7255286/ /pubmed/32565113 http://dx.doi.org/10.1016/j.prrv.2020.05.002 Text en © 2020 Elsevier Ltd. All rights reserved. Since January 2020 Elsevier has created a COVID-19 resource centre with free information in English and Mandarin on the novel coronavirus COVID-19. The COVID-19 resource centre is hosted on Elsevier Connect, the company's public news and information website. Elsevier hereby grants permission to make all its COVID-19-related research that is available on the COVID-19 resource centre - including this research content - immediately available in PubMed Central and other publicly funded repositories, such as the WHO COVID database with rights for unrestricted research re-use and analyses in any form or by any means with acknowledgement of the original source. These permissions are granted for free by Elsevier for as long as the COVID-19 resource centre remains active.
spellingShingle Mini-symposium: CF in the age of modulators (Part I)
Balfour-Lynn, I.M.
King, J.A.
CFTR modulator therapies – Effect on life expectancy in people with cystic fibrosis
title CFTR modulator therapies – Effect on life expectancy in people with cystic fibrosis
title_full CFTR modulator therapies – Effect on life expectancy in people with cystic fibrosis
title_fullStr CFTR modulator therapies – Effect on life expectancy in people with cystic fibrosis
title_full_unstemmed CFTR modulator therapies – Effect on life expectancy in people with cystic fibrosis
title_short CFTR modulator therapies – Effect on life expectancy in people with cystic fibrosis
title_sort cftr modulator therapies – effect on life expectancy in people with cystic fibrosis
topic Mini-symposium: CF in the age of modulators (Part I)
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7255286/
https://www.ncbi.nlm.nih.gov/pubmed/32565113
http://dx.doi.org/10.1016/j.prrv.2020.05.002
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