Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

Patients with homozygous Phe508del cystic fibrosis were assigned to receive combination tezacaftor–ivacaftor or placebo for 24 weeks. The combination resulted in an FEV 1 that was 4 percentage points higher and a pulmonary-exacerbation rate that was 35% lower than with placebo.

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Bibliographische Detailangaben
Veröffentlicht in:	The New England journal of medicine 2017-11, Vol.377 (21), p.2013-2023
Hauptverfasser:	Taylor-Cousar, Jennifer L, Munck, Anne, McKone, Edward F, van der Ent, Cornelis K, Moeller, Alexander, Simard, Christopher, Wang, Linda T, Ingenito, Edward P, McKee, Charlotte, Lu, Yimeng, Lekstrom-Himes, Julie, Elborn, J. Stuart
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Adult Aminophenols - adverse effects Aminophenols - pharmacology Aminophenols - therapeutic use Benzodioxoles - adverse effects Benzodioxoles - pharmacology Benzodioxoles - therapeutic use Body Mass Index Child Clinical trials Cystic fibrosis Cystic Fibrosis - drug therapy Cystic Fibrosis - genetics Cystic Fibrosis - physiopathology Cystic Fibrosis Transmembrane Conductance Regulator - adverse effects Cystic Fibrosis Transmembrane Conductance Regulator - genetics Cystic Fibrosis Transmembrane Conductance Regulator - pharmacology Cystic Fibrosis Transmembrane Conductance Regulator - therapeutic use Double-Blind Method Drug Combinations Drug dosages Drug therapy Female Forced Expiratory Volume - drug effects Homozygote Humans Indoles - adverse effects Indoles - pharmacology Indoles - therapeutic use Male Mutation Patients Pharmaceuticals Proteins Quality of Life Quinolones - adverse effects Quinolones - pharmacology Quinolones - therapeutic use Young Adult
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Beschreibung
Zusammenfassung:	Patients with homozygous Phe508del cystic fibrosis were assigned to receive combination tezacaftor–ivacaftor or placebo for 24 weeks. The combination resulted in an FEV 1 that was 4 percentage points higher and a pulmonary-exacerbation rate that was 35% lower than with placebo.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa1709846