Abstract
Introduction: Lumacaftor/ivacaftor (lum/iva) was introduced in the Netherlands from November 2017 on. Phase 3 trials have shown small, but favorable improvement in lung function, sweat chloride and anthropometry. Our aim was to evaluate the real life safety and efficacy of lum/iva in children aged 6–18 years in the Netherlands during the first year of treatment with emphasis on small airway disease evaluated by CT-scan and multiple breath nitrogen washout.
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