A CFTR Potentiator in Patients with Cystic Fibrosis and the <i>G551D</i> Mutation

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Résumé

BACKGROUND: Increasing the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein is a potential treatment for cystic fibrosis. METHODS: 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)). RESULTS: The change from baseline through week 24 in the percent of predicted FEV(1) 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. Subjects receiving ivacaftor were 55% less likely to have a pulmonary exacerbation than were patients receiving placebo, through week 48 (P<0.001). In addition, through week 48, subjects in the ivacaftor group scored 8.6 points higher than did subjects in the placebo group on the respiratory-symptoms domain of the Cystic Fibrosis Questionnaire-revised instrument (a 100-point scale, with higher numbers indicating a lower effect of symptoms on the patient's quality of life) (P<0.001). By 48 weeks, patients treated with ivacaftor had gained, on average, 2.7 kg more weight than had patients receiving placebo (P<0.001). The change from baseline through week 48 in the concentration of sweat chloride, a measure of CFTR activity, with ivacaftor as compared with placebo was -48.1 mmol per liter (P<0.001). The incidence of adverse events was similar with ivacaftor and placebo, with a lower proportion of serious adverse events with ivacaftor than with placebo (24% vs. 42%). CONCLUSIONS: Ivacaftor was associated with improvements in lung function at 2 weeks that were sustained through 48 weeks. Substantial improvements were also observed in the risk of pulmonary exacerbations, patient-reported respiratory symptoms, weight, and concentration of sweat chloride. (Funded by Vertex Pharmaceuticals and others; VX08-770-102 ClinicalTrials.gov number, NCT00909532.).

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La notice

Revue

New England Journal of Medicine

Thématique

Cystic Fibrosis Research Advances

Domaine

Medicine

Établissements canadiens

Hospital for Sick ChildrenUniversity of TorontoSt. Michael's Hospital

Organismes subventionnaires

National Heart, Lung, and Blood InstituteNational Institutes of HealthChildren's Hospital of PittsburghUniversity of AlabamaNational Center for Research ResourcesUniversity of PennsylvaniaSeattle Children's Research InstituteVertex PharmaceuticalsCase Western Reserve UniversityMedical Center, University of PittsburghCystic Fibrosis FoundationUniversity of PittsburghNational Institute of Diabetes and Digestive and Kidney DiseasesJohns Hopkins UniversityImperial College LondonUniversity of WashingtonCystic Fibrosis Foundation TherapeuticsNational Institute for Health and Care ResearchUniversity of Alabama at BirminghamChildren's Hospital of Philadelphia

Mots-clés

PotentiatorCystic fibrosisMedicineIvacaftorCystic fibrosis transmembrane conductance regulatorMutationInternal medicineGeneticsPharmacology

Résumé présent dans OpenAlex

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