%0 Journal Article
%T Modeling long-term health outcomes of patients with cystic fibrosis homozygous for F508del-CFTR treated with lumacaftor/ivacaftor
%A Alexandra Ward
%A Christopher Pelligra
%A Conor Chandler
%A Jack K. Ishak
%A Jaime L. Rubin
%A Lasair O¡¯Callaghan
%A Michael W. Konstan
%A Theodore G. Liou
%J Archive of "Therapeutic Advances in Respiratory Disease".
%D 2019
%R 10.1177/1753466618820186
%X Lumacaftor/ivacaftor combination therapy is efficacious and generally safe for patients with cystic fibrosis (CF) homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation. However, long-term survival benefits of lumacaftor/ivacaftor (LUM/IVA) cannot yet be quantified. Simulation models can provide predictions about long-term health outcomes. In this study, we aimed to project long-term health outcomes of LUM/IVA plus standard care (SC) in patients with CF homozygous for F508del-CFTR
%K cystic fibrosis
%K ivacaftor
%K lumacaftor
%K percent predicted forced expiratory volume in 1 second (ppFEV1)
%K simulation model
%K survival
%K survival projection
%U https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6366006/