Lumacaftor/Ivacaftor reduces pulmonary exacerbations in patients irrespective of initial changes in FEV 1

• Published in: Journal of cystic fibrosis 2019-01, Vol.18 (1), p.94-101
• Main Authors: McColley, Susanna A, Konstan, Michael W, Ramsey, Bonnie W, Stuart Elborn, J, Boyle, Michael P, Wainwright, Claire E, Waltz, David, Vera-Llonch, Montserrat, Marigowda, Gautham, Jiang, John G, Rubin, Jaime L
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Aged; Aminophenols - therapeutic use; Aminopyridines - therapeutic use; Benzodioxoles - therapeutic use; Child; Cystic Fibrosis - drug therapy; Cystic Fibrosis - physiopathology; Disease Progression; Double-Blind Method; Drug Combinations; Female; Follow-Up Studies; Forced Expiratory Volume - drug effects; Forced Expiratory Volume - physiology; Humans; Lung - drug effects; Lung - physiopathology; Male; Middle Aged; Pulmonary/Respiratory; Quinolones - therapeutic use; Respiratory Function Tests; Retrospective Studies; Treatment Outcome; Young Adult
• ISSN: 1569-1993; 1873-5010
• DOI: 10.1016/j.jcf.2018.07.011

AbstractBackgroundImproved lung function and fewer pulmonary exacerbations (PEx) were observed with lumacaftor/ivacaftor (LUM/IVA) in patients with cystic fibrosis homozygous for F508del. It is unknown whether PEx reduction extends to patients without early lung function improvement. MethodsPost hoc analyses of pooled phase 3 data ( NCT01807923, NCT01807949) categorized LUM/IVA-treated patients by percent predicted forced expiratory volume in 1 s (ppFEV 1) change from baseline to day 15 into threshold categories (absolute change ≤0 vs >0; relative change  0 and 0.85 for those with absolute change ≤0 (respective rate ratios vs placebo [95% CI]: 0.53 [0.40–0.69; P