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 language | English (US) |
|---|---|
| Article number | 93 |
| Journal | Health and Quality of Life Outcomes |
| Volume | 13 |
| Issue number | 1 |
| DOIs | |
| State | Published - Jul 2 2015 |
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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 journal › Article
}
TY - JOUR
T1 - Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation
T2 - Patient-reported outcomes in the STRIVE randomized, controlled trial
AU - Quittner, Alexandra
AU - Suthoff, Ellison
AU - Rendas-Baum, Regina
AU - Bayliss, Martha S.
AU - Sermet-Gaudelus, Isabelle
AU - Castiglione, Brenda
AU - Vera-Llonch, Montserrat
PY - 2015/7/2
Y1 - 2015/7/2
N2 - 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.
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.
KW - CFQ-R
KW - Cystic fibrosis
KW - Health-related quality of life
KW - Ivacaftor
KW - Patient-reported outcomes
UR - http://www.scopus.com/inward/record.url?scp=84936882999&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84936882999&partnerID=8YFLogxK
U2 - 10.1186/s12955-015-0293-6
DO - 10.1186/s12955-015-0293-6
M3 - Article
VL - 13
JO - Health and Quality of Life Outcomes
JF - Health and Quality of Life Outcomes
SN - 1477-7525
IS - 1
M1 - 93
ER -