About: A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation

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About: A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation Goto Sponge Distinct Permalink

An Entity of Type : http://linked.opendata.cz/ontology/domain/vavai/Vysledek, within Data Space : linked.opendata.cz associated with source document(s)

Attributes	Values
rdf:type	skos:Concept http://linked.opendata.cz/ontology/domain/vavai/Vysledek
rdfs:seeAlso	http://www.ncbi.nlm.nih.gov/pubmed/22047557
Description	Increasing the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein is a potential treatment for cystic fibrosis. We conducted a randomized, double-blind, placebo-controlled trial to evaluate ivacaftor (VX-770), a CFTR potentiator, in subjects 12 years of age or older with cystic fibrosis and at least one G551D-CFTR mutation. Subjects were randomly assigned to receive 150 mg of ivacaftor every 12 hours (84 subjects, of whom 83 received at least one dose) or placebo (83, of whom 78 received at least one dose) for 48 weeks. The primary end point was the estimated mean change from baseline through week 24 in the percent of predicted forced expiratory volume in 1 second (FEV(1)). The change from baseline through week 24 in the percent of predicted FEV1 was greater by 10.6 percentage points in the ivacaftor group than in the placebo group (P < 0.001). Effects on pulmonary function were noted by 2 weeks, and a significant treatment effect was maintained through week 48. Increasing the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein is a potential treatment for cystic fibrosis. We conducted a randomized, double-blind, placebo-controlled trial to evaluate ivacaftor (VX-770), a CFTR potentiator, in subjects 12 years of age or older with cystic fibrosis and at least one G551D-CFTR mutation. Subjects were randomly assigned to receive 150 mg of ivacaftor every 12 hours (84 subjects, of whom 83 received at least one dose) or placebo (83, of whom 78 received at least one dose) for 48 weeks. The primary end point was the estimated mean change from baseline through week 24 in the percent of predicted forced expiratory volume in 1 second (FEV(1)). The change from baseline through week 24 in the percent of predicted FEV1 was greater by 10.6 percentage points in the ivacaftor group than in the placebo group (P < 0.001). Effects on pulmonary function were noted by 2 weeks, and a significant treatment effect was maintained through week 48. (en)
Title	A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation (en)
skos:prefLabel	A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation (en)
skos:notation	RIV/00064203:_____/11:7160!RIV12-MZ0-00064203
http://linked.open...avai/riv/aktivita	I
http://linked.open...avai/riv/aktivity	I
http://linked.open...iv/cisloPeriodika	18
http://linked.open...vai/riv/dodaniDat	2012
http://linked.open...aciTvurceVysledku	Dřevínek, Pavel
http://linked.open.../riv/druhVysledku	J - Článek v odborném periodiku
http://linked.open...iv/duvernostUdaju	S - Úplné a pravdivé údaje nepodléhající ochraně podle zvláštních právních předpisů
http://linked.open...titaPredkladatele	Fakultní nemocnice v Motole
http://linked.open...dnocenehoVysledku	183653
http://linked.open...ai/riv/idVysledku	RIV/00064203:_____/11:7160
http://linked.open...riv/jazykVysledku	eng - angličtina
http://linked.open.../riv/klicovaSlova	randomized controlled-trial; pseudomonas-aeruginosa; pulmonary-function; identification; questionnaire; azithromycin; mechanisms; guidelines; health; vx-770 (en)
http://linked.open.../riv/klicoveSlovo	health identification pulmonary-function randomized controlled-trial azithromycin guidelines mechanisms pseudomonas-aeruginosa questionnaire vx-770
http://linked.open...odStatuVydavatele	US - Spojené státy americké
http://linked.open...ontrolniKodProRIV	[750550BD00B9]
http://linked.open...i/riv/nazevZdroje	New England Journal of Medicine
http://linked.open...in/vavai/riv/obor	FP
http://linked.open...ichTvurcuVysledku	1 (xsd:int)
http://linked.open...cetTvurcuVysledku	19 (xsd:int)
http://linked.open...UplatneniVysledku	2011
http://linked.open...v/svazekPeriodika	365
http://linked.open...iv/tvurceVysledku	Bell, SC Davies, J. Dong, QM Dřevínek, Pavel Elborn, J. S. Griese, M. Konstan, MW McElvaney, NG McKone, EF Moss, R. Ordonez, C. Ramsey, BW Ratjen, F. Rodriguez, S. Rowe, S. M. Sermet-Gaudelus, I. Tullis, E. Wainwright, CE Yen, K.
http://linked.open...ain/vavai/riv/wos	000296424200005
issn	0028-4793
number of pages	10 (xsd:int)

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