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

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.

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Déclaration de conflit d'intérêts

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.