Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR.

Steven M RoweSusanna A McColleyErnst RietschelXiaolei LiScott C BellMichael W KonstanGautham MarigowdaDavid WaltzMichael P Boylenull null

Published in: Annals of the American Thoracic Society (2018)

Sweat chloride and respiratory symptom scores improved with lumacaftor/ivacaftor, though no meaningful benefit was seen in ppFEV1 or body mass index in patients heterozygous for F508del-CFTR. Clinical trial registered with www.clinicaltrials.gov (NCT01225211).

Keyphrases

cystic fibrosis
body mass index
clinical trial
end stage renal disease
early onset
ejection fraction
newly diagnosed
prognostic factors
peritoneal dialysis
randomized controlled trial
patient reported
physical activity
study protocol
double blind
weight loss
respiratory tract