REAL‐world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations—A case report

This study summarizes efficacy of ivacaftor treatment in 2 infants in a real‐world setting. A distinct decline of sweat chloride and lung clearance index plus increase in fecal elastase was seen. The results underline the early and sustainable effect and give cause for discussing whether a reduction...

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Autores principales: Fuchs, Teresa, Appelt, Dorothea, Niedermayr, Katharina, Ellemunter, Helmut
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Case Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8829656/
https://www.ncbi.nlm.nih.gov/pubmed/35154718
http://dx.doi.org/10.1002/ccr3.5364
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author Fuchs, Teresa
Appelt, Dorothea
Niedermayr, Katharina
Ellemunter, Helmut
author_facet Fuchs, Teresa
Appelt, Dorothea
Niedermayr, Katharina
Ellemunter, Helmut
author_sort Fuchs, Teresa
collection PubMed
description This study summarizes efficacy of ivacaftor treatment in 2 infants in a real‐world setting. A distinct decline of sweat chloride and lung clearance index plus increase in fecal elastase was seen. The results underline the early and sustainable effect and give cause for discussing whether a reduction in standard cystic fibrosis therapy is possible.
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spelling pubmed-88296562022-02-11 REAL‐world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations—A case report Fuchs, Teresa Appelt, Dorothea Niedermayr, Katharina Ellemunter, Helmut Clin Case Rep Case Reports This study summarizes efficacy of ivacaftor treatment in 2 infants in a real‐world setting. A distinct decline of sweat chloride and lung clearance index plus increase in fecal elastase was seen. The results underline the early and sustainable effect and give cause for discussing whether a reduction in standard cystic fibrosis therapy is possible. John Wiley and Sons Inc. 2022-02-10 /pmc/articles/PMC8829656/ /pubmed/35154718 http://dx.doi.org/10.1002/ccr3.5364 Text en © 2022 The Authors. Clinical Case Reports published by John Wiley & Sons Ltd. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.
spellingShingle Case Reports
Fuchs, Teresa
Appelt, Dorothea
Niedermayr, Katharina
Ellemunter, Helmut
REAL‐world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations—A case report
title REAL‐world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations—A case report
title_full REAL‐world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations—A case report
title_fullStr REAL‐world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations—A case report
title_full_unstemmed REAL‐world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations—A case report
title_short REAL‐world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations—A case report
title_sort real‐world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations—a case report
topic Case Reports
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8829656/
https://www.ncbi.nlm.nih.gov/pubmed/35154718
http://dx.doi.org/10.1002/ccr3.5364
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