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Clinical Trial
. 2023 Jan;22(1):119-123.
doi: 10.1016/j.jcf.2022.08.018. Epub 2022 Sep 14.

Non-respiratory health-related quality of life in people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor

Isabelle Fajac  1 , Cori Daines  2 , Isabelle Durieu  3 , Jennifer L Goralski  4 , Harry Heijerman  5 , Christiane Knoop  6 , Christof Majoor  7 , Bote G Bruinsma  8 , Samuel Moskowitz  9 , Valentin Prieto-Centurion  10 , Kate Van Brunt  11 , Yaohua Zhang  12 , Alexandra Quittner  13
Affiliations
Free article
Clinical Trial

Non-respiratory health-related quality of life in people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor

Isabelle Fajac et al. J Cyst Fibros. 2023 Jan.
Free article

Abstract

Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in people with cystic fibrosis (CF) heterozygous for F508del and a minimal function mutation (F/MF) or homozygous for F508del (F/F) in two pivotal Phase 3 trials, significantly improving percentage predicted forced expiratory volume in 1 second, Cystic Fibrosis Questionnaire-Revised, Respiratory Domain (CFQ-R RD) scores, and sweat chloride concentration. Here, we analyzed the 11 non-respiratory domains (non-RDs) of the CFQ-R, which assess general health-related quality of life (i.e., Physical Functioning, Role Functioning, Vitality, Health Perceptions, Emotional Functioning, and Social Functioning) and quality of life impacted by CF (i.e., Body Image, Eating Problems, Treatment Burden, Weight, and Digestive Symptoms), for participants in these two Phase 3 trials. ELX/TEZ/IVA treatment led to higher scores in all CFQ-R non-RDs, with improvements in most domains compared with control treatments. These findings demonstrate that ELX/TEZ/IVA improves a range of CF-specific symptoms and general functioning and well-being.

Keywords: CFQ-R; Cystic fibrosis; Elexacaftor/tezacaftor/ivacaftor; Quality of life.

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Conflict of interest statement

Declaration of Competing Interest IF reports grants and personal fees from Vertex during the conduct of the study. KVB, BGB, VP-C, and YZ are employees of Vertex and may own stock or stock options in the company. SMM was an employee at Vertex Pharmaceuticals at the time of the study. HH reports personal fees from Vertex, Gilead, Horizon, PTC, and Chiesi and serves on advisory boards for Vertex and PTC, outside the submitted work. SM has a patent pending for Methods of Treatment for Cystic Fibrosis, a patent pending for Methods of Treatment of Cystic Fibrosis, and a patent pending for Pharmaceutical Compositions for Treating Cystic Fibrosis. AQ reports consulting fees from Vertex and Insmed, outside of the submitted work. CD, ID, JG, CK, and CM have no disclosures to report.

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