Article
Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial.
Health and quality of life outcomes - 2 Jul 2015
Quittner Alexandra, Suthoff Ellison, Rendas-Baum Regina, Bayliss Martha S, Sermet-Gaudelus Isabelle, Castiglione Brenda, Vera-Llonch Montserrat
Abstract excerpt
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,...
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