Lumacaftor/Ivacaftor reduces pulmonary exacerbations in patients irrespective of initial changes in FEV1

Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
Susanna A McColleyJaime L Rubin

Abstract

Improved 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. Post hoc analyses of pooled phase 3 data (NCT01807923, NCT01807949) categorized LUM/IVA-treated patients by percent predicted forced expiratory volume in 1 s (ppFEV1) change from baseline to day 15 into threshold categories (absolute change ≤0 vs >0; relative change <5% vs ≥5%) and compared PEx rates vs placebo. LUM (400 mg q12h)/IVA (250 mg q12h)-treated patients (n = 369) experienced significantly fewer PEx vs placebo, regardless of threshold category. With LUM/IVA, PEx rate per patient per year was 0.60 for those with absolute change in ppFEV1 > 0 and 0.85 for those with absolute change ≤0 (respective rate ratios vs placebo [95% CI]: 0.53 [0.40-0.69; P < .0001], 0.74 [0.55-0.99; P = .04]). LUM/IVA significantly reduced PEx, even in patients without early lung function improvement.

Citations

Oct 12, 2019·American Journal of Respiratory and Critical Care Medicine·Pierre-Régis BurgelUNKNOWN French Cystic Fibrosis Reference Network Study Group
Aug 25, 2020·Pediatric Pulmonology·Adrienne P Savant, Susanna A McColley
Mar 22, 2020·European Respiratory Review : an Official Journal of the European Respiratory Society·J Stuart Elborn
Mar 11, 2020·Frontiers in Pharmacology·Miquéias Lopes-Pacheco
Jan 20, 2021·Journal of Medical Internet Research·Andreas HagerCarolina Wannheden
Dec 19, 2019·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Koliarne TongPeter Wark
Mar 14, 2021·Molecular Genetics & Genomic Medicine·Vito TerlizziGiovanni Taccetti
Jun 5, 2021·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Scott H DonaldsonWilliam D Bennett
Jul 24, 2021·European Archives of Oto-rhino-laryngology : Official Journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : Affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery·Joanna KrajewskaTomasz Zatoński
May 6, 2021·American Journal of Respiratory and Critical Care Medicine·Pierre-Régis BurgelClémence Martin

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