Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation

Patient-reported outcomes in the STRIVE randomized, controlled trial

Alexandra Quittner, Ellison Suthoff, Regina Rendas-Baum, Martha S. Bayliss, Isabelle Sermet-Gaudelus, Brenda Castiglione, Montserrat Vera-Llonch

Research output: Contribution to journalArticle

17 Citations (Scopus)

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.

Original languageEnglish (US)
Article number93
JournalHealth and Quality of Life Outcomes
Volume13
Issue number1
DOIs
StatePublished - Jul 2 2015

Fingerprint

Cystic Fibrosis
Randomized Controlled Trials
Mutation
Therapeutics
ivacaftor
Patient Reported Outcome Measures
Placebos
Premature Mortality
Body Image
Health
Eating
Morbidity

Keywords

  • CFQ-R
  • Cystic fibrosis
  • Health-related quality of life
  • Ivacaftor
  • Patient-reported outcomes

ASJC Scopus subject areas

  • Public Health, Environmental and Occupational Health
  • Medicine(all)

Cite this

Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation : Patient-reported outcomes in the STRIVE randomized, controlled trial. / Quittner, Alexandra; Suthoff, Ellison; Rendas-Baum, Regina; Bayliss, Martha S.; Sermet-Gaudelus, Isabelle; Castiglione, Brenda; Vera-Llonch, Montserrat.

In: Health and Quality of Life Outcomes, Vol. 13, No. 1, 93, 02.07.2015.

Research output: Contribution to journalArticle

Quittner, Alexandra ; Suthoff, Ellison ; Rendas-Baum, Regina ; Bayliss, Martha S. ; Sermet-Gaudelus, Isabelle ; Castiglione, Brenda ; Vera-Llonch, Montserrat. / Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation : Patient-reported outcomes in the STRIVE randomized, controlled trial. In: Health and Quality of Life Outcomes. 2015 ; Vol. 13, No. 1.
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AU - Rendas-Baum, Regina

AU - Bayliss, Martha S.

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AU - Vera-Llonch, Montserrat

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AB - 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.

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