AustralieFrV1, Main, Exploration, bibRecord, 004F96

Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with a G551D Mutation

Identifieur interne : 004F96 ( Main/Exploration ); précédent : 004F95; suivant : 004F97

Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with a G551D Mutation

Auteurs : Jane C. Davies [Royaume-Uni] ; Claire E. Wainwright [Australie] ; Gerard J. Canny [Irlande (pays)] ; Mark A. Chilvers [Canada] ; Michelle S. Howenstine [États-Unis] ; Anne Munck [France] ; Jochen G. Mainz [Allemagne] ; Sally Rodriguez [États-Unis] ; HAIHONG LI [États-Unis] ; Karl Yen [États-Unis] ; Claudia L. Ordonez [États-Unis] ; Richard Ahrens [États-Unis]

Source :

American journal of respiratory and critical care medicine [ 1073-449X ] ; 2013.

RBID : Pascal:13-0223598

Descripteurs français

Pascal (Inist)
- Mucoviscidose, Pathologie de l'appareil respiratoire, Efficacité, Toxicité, Sécurité, Homme, Malade, Personne âgée, Mutation, Régulateur conductance transmembranaire mucoviscidose, Protéine, Poumon, Sueur, Réanimation, Soin intensif, Ivacaftor.
Wicri :
- topic : Homme, Personne âgée.

English descriptors

KwdEn :
- Cystic fibrosis, Cystic fibrosis transmembrane conductance regulator, Efficiency, Elderly, Human, Intensive care, Ivacaftor, Lung, Mutation, Patient, Protein, Respiratory disease, Resuscitation, Safety, Sweat, Toxicity.

Abstract

Rationale: Ivacaftor (VX-770), a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, has been shown to improve lung function, pulmonary exacerbation rate, respiratory symptoms, and weight gain compared with placebo in patients with cystic fibrosis aged 12 years or older with a G551D-CFTR mutation. Objectives: This randomized, double-blind, placebo-controlled trial evaluated ivacaftor in patients with cystic fibrosis aged 6-11 years with a G551D-CFTR mutation on at least one allele. Methods: Patients were randomly assigned to receive ivacaftor administered orally at 150 mg (n = 26) or placebo (n = 26) every 12 hours for 48 weeks in addition to existing prescribed cystic fibrosis therapies. Measurements and Main Results: Despite near-normal mean baseline values in FEV₁, patients receiving ivacaftor had a significant increase in percent predicted FEV₁ from baseline through Week 24 versus placebo group (treatment effect, 12.5 percentage points; P < 0.001). Effects on pulmonary function were evident by 2 weeks, and a significant treatment effect was maintained through Week 48. Patients treated with ivacaftor gained, on average, 2.8 kg more than those receiving placebo at Week 48 (P < 0.001). The change from baseline through Week 48 in the concentration of sweat chloride, a measure of CFTR activity, with ivacaftor was -53.5 mmol/L (P<0.001) versus placebo. The incidence of adverse events was similar in the two groups. Conclusions: In patients who are younger and healthier than those in previously studied populations, ivacaftor demonstrated a significant improvement in pulmonary function, weight, and CFTR activity compared with placebo. Clinical trial registered with www.clinicaltrials.gov (NCT00909727).

Url:

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3734608

Affiliations:

Allemagne, Australie, Canada, France, Irlande (pays), Royaume-Uni, États-Unis
Angleterre, Grand Londres, Indiana, Iowa, Massachusetts, Île-de-France
Iowa City, Londres, Paris
Université de l'Iowa

Le document en format XML

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<series><title level="j" type="main">American journal of respiratory and critical care medicine</title>
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<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Cystic fibrosis</term>
<term>Cystic fibrosis transmembrane conductance regulator</term>
<term>Efficiency</term>
<term>Elderly</term>
<term>Human</term>
<term>Intensive care</term>
<term>Ivacaftor</term>
<term>Lung</term>
<term>Mutation</term>
<term>Patient</term>
<term>Protein</term>
<term>Respiratory disease</term>
<term>Resuscitation</term>
<term>Safety</term>
<term>Sweat</term>
<term>Toxicity</term>
</keywords>
<keywords scheme="Pascal" xml:lang="fr"><term>Mucoviscidose</term>
<term>Pathologie de l'appareil respiratoire</term>
<term>Efficacité</term>
<term>Toxicité</term>
<term>Sécurité</term>
<term>Homme</term>
<term>Malade</term>
<term>Personne âgée</term>
<term>Mutation</term>
<term>Régulateur conductance transmembranaire mucoviscidose</term>
<term>Protéine</term>
<term>Poumon</term>
<term>Sueur</term>
<term>Réanimation</term>
<term>Soin intensif</term>
<term>Ivacaftor</term>
</keywords>
<keywords scheme="Wicri" type="topic" xml:lang="fr"><term>Homme</term>
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<front><div type="abstract" xml:lang="en">Rationale: Ivacaftor (VX-770), a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, has been shown to improve lung function, pulmonary exacerbation rate, respiratory symptoms, and weight gain compared with placebo in patients with cystic fibrosis aged 12 years or older with a G551D-CFTR mutation. Objectives: This randomized, double-blind, placebo-controlled trial evaluated ivacaftor in patients with cystic fibrosis aged 6-11 years with a G551D-CFTR mutation on at least one allele. Methods: Patients were randomly assigned to receive ivacaftor administered orally at 150 mg (n = 26) or placebo (n = 26) every 12 hours for 48 weeks in addition to existing prescribed cystic fibrosis therapies. Measurements and Main Results: Despite near-normal mean baseline values in FEV<sub>1</sub>
, patients receiving ivacaftor had a significant increase in percent predicted FEV<sub>1</sub>
 from baseline through Week 24 versus placebo group (treatment effect, 12.5 percentage points; P < 0.001). Effects on pulmonary function were evident by 2 weeks, and a significant treatment effect was maintained through Week 48. Patients treated with ivacaftor gained, on average, 2.8 kg more than those receiving placebo at Week 48 (P < 0.001). The change from baseline through Week 48 in the concentration of sweat chloride, a measure of CFTR activity, with ivacaftor was -53.5 mmol/L (P<0.001) versus placebo. The incidence of adverse events was similar in the two groups. Conclusions: In patients who are younger and healthier than those in previously studied populations, ivacaftor demonstrated a significant improvement in pulmonary function, weight, and CFTR activity compared with placebo. Clinical trial registered with www.clinicaltrials.gov (NCT00909727).</div>
</front>
</TEI>
<affiliations><list><country><li>Allemagne</li>
<li>Australie</li>
<li>Canada</li>
<li>France</li>
<li>Irlande (pays)</li>
<li>Royaume-Uni</li>
<li>États-Unis</li>
</country>
<region><li>Angleterre</li>
<li>Grand Londres</li>
<li>Indiana</li>
<li>Iowa</li>
<li>Massachusetts</li>
<li>Île-de-France</li>
</region>
<settlement><li>Iowa City</li>
<li>Londres</li>
<li>Paris</li>
</settlement>
<orgName><li>Université de l'Iowa</li>
</orgName>
</list>
<tree><country name="Royaume-Uni"><region name="Angleterre"><name sortKey="Davies, Jane C" sort="Davies, Jane C" uniqKey="Davies J" first="Jane C." last="Davies">Jane C. Davies</name>
</region>
<name sortKey="Davies, Jane C" sort="Davies, Jane C" uniqKey="Davies J" first="Jane C." last="Davies">Jane C. Davies</name>
</country>
<country name="Australie"><noRegion><name sortKey="Wainwright, Claire E" sort="Wainwright, Claire E" uniqKey="Wainwright C" first="Claire E." last="Wainwright">Claire E. Wainwright</name>
</noRegion>
</country>
<country name="Irlande (pays)"><noRegion><name sortKey="Canny, Gerard J" sort="Canny, Gerard J" uniqKey="Canny G" first="Gerard J." last="Canny">Gerard J. Canny</name>
</noRegion>
</country>
<country name="Canada"><noRegion><name sortKey="Chilvers, Mark A" sort="Chilvers, Mark A" uniqKey="Chilvers M" first="Mark A." last="Chilvers">Mark A. Chilvers</name>
</noRegion>
</country>
<country name="États-Unis"><region name="Indiana"><name sortKey="Howenstine, Michelle S" sort="Howenstine, Michelle S" uniqKey="Howenstine M" first="Michelle S." last="Howenstine">Michelle S. Howenstine</name>
</region>
<name sortKey="Ahrens, Richard" sort="Ahrens, Richard" uniqKey="Ahrens R" first="Richard" last="Ahrens">Richard Ahrens</name>
<name sortKey="Haihong Li" sort="Haihong Li" uniqKey="Haihong Li" last="Haihong Li">HAIHONG LI</name>
<name sortKey="Ordonez, Claudia L" sort="Ordonez, Claudia L" uniqKey="Ordonez C" first="Claudia L." last="Ordonez">Claudia L. Ordonez</name>
<name sortKey="Rodriguez, Sally" sort="Rodriguez, Sally" uniqKey="Rodriguez S" first="Sally" last="Rodriguez">Sally Rodriguez</name>
<name sortKey="Yen, Karl" sort="Yen, Karl" uniqKey="Yen K" first="Karl" last="Yen">Karl Yen</name>
</country>
<country name="France"><region name="Île-de-France"><name sortKey="Munck, Anne" sort="Munck, Anne" uniqKey="Munck A" first="Anne" last="Munck">Anne Munck</name>
</region>
</country>
<country name="Allemagne"><noRegion><name sortKey="Mainz, Jochen G" sort="Mainz, Jochen G" uniqKey="Mainz J" first="Jochen G." last="Mainz">Jochen G. Mainz</name>
</noRegion>
</country>
</tree>
</affiliations>
</record>

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Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with a G551D Mutation

Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with a G551D Mutation

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