Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional study.

Bell, Scott C; Mainz, Jochen G; MacGregor, Gordon; Madge, Susan; Macey, Julie; Fridman, Moshe; Suthoff, Ellison D; Narayanan, Siva; Kinnman, Nils

Bell, Scott C; Mainz, Jochen G; MacGregor, Gordon; Madge, Susan; Macey, Julie; Fridman, Moshe; Suthoff, Ellison D; Narayanan, Siva; Kinnman, Nils.

Affiliation

Bell SC; Department of Thoracic Medicine, The Prince Charles Hospital and QIMR Berghofer Medical Research Institute, Brisbane, QLD, Australia. scott.bell@qimrberghofer.edu.au.
Mainz JG; Jena University Hospital, Jena, Germany.
MacGregor G; Brandenburg Medical School (MHB), University , Brandenburg an der Havel, Germany.
Madge S; Gartnavel General Hospital, Glasgow, UK.
Macey J; Royal Brompton Hospital, London, UK.
Fridman M; University Hospital Bordeaux, Bordeaux, France.
Suthoff ED; AMF Consulting, Los Angeles, CA, USA.
Narayanan S; Formerly of Vertex Pharmaceuticals Incorporated, Boston, MA, USA.
Kinnman N; Decision Resources Group, Burlington, MA, USA.

BMC Pulm Med ; 19(1): 146, 2019 Aug 13.

Article in En | MEDLINE | ID: mdl-31409396

Subject(s)

Aminophenols/therapeutic use; Aminopyridines/therapeutic use; Benzodioxoles/therapeutic use; Cystic Fibrosis Transmembrane Conductance Regulator/genetics; Cystic Fibrosis/drug therapy; Cystic Fibrosis/genetics; Quinolones/therapeutic use; Child; Cross-Sectional Studies; Drug Combinations; Female; Forced Expiratory Volume; Humans; Internationality; Male; Multivariate Analysis; Mutation; Patient Reported Outcome Measures; Quality of Life; Regression Analysis; Surveys and Questionnaires

Key words

Cystic fibrosis; G551D-CFTR; Ivacaftor; Patient-reported outcomes; Work productivity

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Quinolones / Cystic Fibrosis Transmembrane Conductance Regulator / Cystic Fibrosis / Benzodioxoles / Aminophenols / Aminopyridines Type of study: Clinical_trials / Diagnostic_studies / Observational_studies / Prevalence_studies / Risk_factors_studies Limits: Child / Female / Humans / Male Language: En Journal: BMC Pulm Med Year: 2019 Type: Article Affiliation country: Australia

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