Respiratory medicine · 2026
Real-world impact of elexacaftor/tezacaftor/ivacaftor on health-related quality of life in adults with cystic fibrosis: A 12-month multicenter cohort study.
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Kharraz Razan, Tullis Elizabeth, Sykes Jenna, Ma Xiayi, Sandhu Akshdeep, Gryz Zosia, Shulman Yonatan, Tam Julian, Love Allison, Ratjen Felix, Rayment Jonathan H, Quon Bradley S, Stephenson Anne L
Аннотация
BACKGROUND: Elexacaftor/tezacaftor/ivacaftor (ETI) has demonstrated significant improvements in lung function, body mass index (BMI), symptom control, and health-related quality of life (HRQoL) in individuals with cystic fibrosis (CF). However, real-world evidence regarding its long-term impact on HRQoL, particularly in people without significant lung function improvement, remains limited.
OBJECTIVES: This study aimed to (1) assess HRQoL changes across multiple domains in individuals initiating ETI and (2) explore the relationship between clinical outcomes and HRQoL, particularly in those who do not experience a significant improvement in lung function.
METHODS: We conducted a prospective cohort study of adults initiating ETI at CF clinics across Canada. HRQoL was assessed using the Cystic Fibrosis Questionnaire-Revised (CFQ-R), Cystic Fibrosis Quality-of-Life Evaluative Self-Administered Test (CF-QUEST), and Sino-Nasal Outcome Test (SNOT-22) at baseline and at 3, 6, 9, and 12 months post-initiation. Lung function (percent predicted forced expiratory volume in 1 s [ppFEV1]) and BMI were also monitored. Individuals were classified as lung function responders (≥5% increase in ppFEV1) or non-responders (<5% increase).
RESULTS: Among 353 individuals, significant improvements in CFQ-R, CF-QUEST, and SNOT-22 scores were observed within one month of ETI initiation and sustained over 12 months. Notably, both lung function responders and non-responders experienced significant HRQoL improvements, with no evidence of a difference between groups in terms of mean changes in CFQ-R respiratory scores (28.8 vs. 24.2, p = 0.2), CF-QUEST global scores (14.8 vs. 13.0, p = 0.48), or SNOT-22 scores (-13.8 vs. -13.7, p = 0.97).
CONCLUSION: Our real-world data involving a large cohort of PwCF demonstrate that HRQoL improvements are sustained across multiple domains regardless of lung function response. These findings underscore the importance of using a multimodal approach to evaluate the effectiveness of modulator therapy in people with CF.
OBJECTIVES: This study aimed to (1) assess HRQoL changes across multiple domains in individuals initiating ETI and (2) explore the relationship between clinical outcomes and HRQoL, particularly in those who do not experience a significant improvement in lung function.
METHODS: We conducted a prospective cohort study of adults initiating ETI at CF clinics across Canada. HRQoL was assessed using the Cystic Fibrosis Questionnaire-Revised (CFQ-R), Cystic Fibrosis Quality-of-Life Evaluative Self-Administered Test (CF-QUEST), and Sino-Nasal Outcome Test (SNOT-22) at baseline and at 3, 6, 9, and 12 months post-initiation. Lung function (percent predicted forced expiratory volume in 1 s [ppFEV1]) and BMI were also monitored. Individuals were classified as lung function responders (≥5% increase in ppFEV1) or non-responders (<5% increase).
RESULTS: Among 353 individuals, significant improvements in CFQ-R, CF-QUEST, and SNOT-22 scores were observed within one month of ETI initiation and sustained over 12 months. Notably, both lung function responders and non-responders experienced significant HRQoL improvements, with no evidence of a difference between groups in terms of mean changes in CFQ-R respiratory scores (28.8 vs. 24.2, p = 0.2), CF-QUEST global scores (14.8 vs. 13.0, p = 0.48), or SNOT-22 scores (-13.8 vs. -13.7, p = 0.97).
CONCLUSION: Our real-world data involving a large cohort of PwCF demonstrate that HRQoL improvements are sustained across multiple domains regardless of lung function response. These findings underscore the importance of using a multimodal approach to evaluate the effectiveness of modulator therapy in people with CF.