Thomas Keens, Veena Hoffman, Ia Topuria, Ken Elder, Shannon Cerf, Kyra Mulder, Jon Roberts, Jerimiah Lysinger, Maria Del Carmen Reyes, Maria Berdella, Anne Marie Cairns, Manu Jain, Vaidyanathan Ganapathy, Yiyue Lou, Bassem Morcos, Chuntao Wu, Laura Sass
BACKGROUND: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to be safe and efficacious in people with cystic fibrosis (CF) aged ≥2 years. Here, we describe results from an observational study assessing change in burden of illness following initiating ELX/TEZ/IVA in real-world settings. METHODS: This US-based, multicenter, observational study used data from electronic medical records to evaluate real-world burden of illness before and after ELX/TEZ/IVA initiation in people with CF aged ≥12 years heterozygous for F508del and a minimal function mutation ( F /MF) or an uncharacterized CFTR mutation...
April 15, 2024: Heliyon