Introduction of Ivacaftor/Lumacaftor in Children With Cystic Fibrosis Homozygous for F508del in the Netherlands: A Nationwide Real-Life Study.

ABSTRACT:

Introduction

Lumacaftor/ivacaftor (lum/iva) was introduced in the Netherlands in 2017. We investigated 1-year efficacy of lum/iva on lung function and small airway and structural lung disease evaluated by multiple breath nitrogen washout and CT scan. Additionally, we investigated effects of lum/iva on exacerbations, anthropometry, sweat chloride and safety in children with CF in the Netherlands.

Methods

Children with CF aged 6-18 years and homozygous for F508del treated in one of the seven Dutch CF centers for at least 12 months were eligible. Data were extracted from the Dutch CF Registry and electronic patient records. Primary outcome was change in percent predicted FEV₁ (ppFEV₁) after 12 months.

Results

Nationwide, 247 children with CF were el

SUBMITTER: Zwitserloot AM 

PROVIDER: S-EPMC11715133 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature