title: Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial.
abstract: BACKGROUND
  Cystic fibrosis (CF) is an inherited, rare autosomal recessive disease that results in chronically debilitating morbidities and high premature mortality. We evaluated how ivacaftor treatment affected CF symptoms, functioning, and well-being, as measured by the Cystic Fibrosis Questionnaire-Revised (CFQ-R), a widely-used patient-reported outcome (PRO) measure.
  
  
  METHODS
  STRIVE, a double-blind, placebo-controlled randomized trial, evaluated ivacaftor (150 mg) in CF patients aged 12+ with the G551D-CFTR mutation for 48 weeks. Treatment effect analysis used a mixed-effects repeated measures model. Treatment benefit analyses applied the cumulative distribution function and a categorical analysis of change scores ("improvement," "no change," or "decline"). Content-based interpretation examined treatment effect on specific item responses.
  
  
  RESULTS
  Data from 152 patients with a baseline CFQ-R assessment were analyzed. The treatment effect analysis favored treatment with ivacaftor over placebo on the Body Image, Eating, Health Perceptions, Physical Functioning, Respiratory, Social Functioning, Treatment Burden, and Vitality scales. Findings were supported by the analysis of categorical change. On all CFQ-R scales, the percentage of patients who improved was greater for ivacaftor. In the content-based analysis, the treatment benefit was characterized by better scores across a broad range of domains.
  
  
  CONCLUSIONS
  Results illustrate broad benefits of ivacaftor treatment across many domains: respiratory symptoms, physical and social functioning, health perceptions, and vitality, as measured by the CFQ-R. The breadth of improvements reflects the systemic mechanism of action of ivacaftor compared to other therapies. Findings support the patient-reported value of ivacaftor treatment in this patient population.
  
  
  BACKGROUND
  ClinicalTrials.gov NCT00909532.
authors.affiliation: Department of Psychology, University of Miami, 5665 Ponce de Leon Blvd, Coral Gables, FL, 33146, USA. aquittner@miami.edu.
authors.forename: Alexandra
authors.initials: A
authors.lastname: Quittner
authors.affiliation: Vertex Pharmaceuticals Incorporated, 50 Northern Avenue, Boston, MA, 02210, USA. ellison_suthoff@vrtx.com.
authors.forename: Ellison
authors.initials: E
authors.lastname: Suthoff
authors.affiliation: Optum, 24 Albion Road, Bldg 400, Lincoln, RI, 02854, USA. rrbaum@qualitymetric.com.
authors.forename: Regina
authors.initials: R
authors.lastname: Rendas-Baum
authors.affiliation: Optum, 24 Albion Road, Bldg 400, Lincoln, RI, 02854, USA. mbayliss@qualitymetric.com.
authors.forename: Martha S
authors.initials: MS
authors.lastname: Bayliss
authors.affiliation: Hopital Necker Enfants Malades, Universite Paris Sorbonne, 149 Rue de Sèvres, Paris, 75015, France. isabelle.sermet@nck.aphp.fr.
authors.forename: Isabelle
authors.initials: I
authors.lastname: Sermet-Gaudelus
authors.affiliation: Vertex Pharmaceuticals Incorporated, 50 Northern Avenue, Boston, MA, 02210, USA. brenda_castiglione@vrtx.com.
authors.forename: Brenda
authors.initials: B
authors.lastname: Castiglione
authors.affiliation: Vertex Pharmaceuticals Incorporated, 50 Northern Avenue, Boston, MA, 02210, USA. montserrat_vera-llonch@vrtx.com.
authors.forename: Montserrat
authors.initials: M
authors.lastname: Vera-Llonch
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000627:Aminophenols / Q000008:administration & dosage*; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000235:genetics*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D058990:Molecular Targeted Therapy / Q000379:methods*; D009154:Mutation; D063868:Patient Outcome Assessment; D011788:Quality of Life; D015363:Quinolones / Q000008:administration & dosage*; D055815:Young Adult
chemical_list: D000627:Aminophenols; C505032:CFTR protein, human; D015363:Quinolones; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; C545203:ivacaftor