TY - JOUR
T1 - Non-respiratory health-related quality of life in people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor
AU - Fajac, Isabelle
AU - Daines, Cori
AU - Durieu, Isabelle
AU - Goralski, Jennifer L
AU - Heijerman, Harry
AU - Knoop, Christiane
AU - Majoor, Christof
AU - Bruinsma, Bote G
AU - Moskowitz, Samuel
AU - Prieto-Centurion, Valentin
AU - Van Brunt, Kate
AU - Zhang, Yaohua
AU - Quittner, Alexandra
N1 - Funding Information:
Editorial coordination and support were provided by Wayne Dunlap, PhD, of Vertex Pharmaceuticals Incorporated. Editorial assistance was provided by Lee Kempster, PhD, of MediTech Media (UK), under the guidance of the authors and was supported by Vertex Pharmaceuticals Incorporated.
Publisher Copyright:
© 2022 The Authors
PY - 2023/1
Y1 - 2023/1
N2 - 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.
AB - 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.
KW - CFQ-R
KW - Cystic fibrosis
KW - Elexacaftor/tezacaftor/ivacaftor
KW - Quality of life
UR - http://www.scopus.com/inward/record.url?scp=85138080309&partnerID=8YFLogxK
U2 - 10.1016/j.jcf.2022.08.018
DO - 10.1016/j.jcf.2022.08.018
M3 - Article
C2 - 36114142
SN - 1569-1993
VL - 22
SP - 119
EP - 123
JO - Journal of Cystic Fibrosis
JF - Journal of Cystic Fibrosis
IS - 1
ER -