Annals of the American Thoracic Society · 2026
Pulmonary Outcomes in People with Cystic Fibrosis who Stop Chronic Daily Therapies: The HERO-2 Study.
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Brown Cynthia D, Psoter Kevin J, Sabadosa Kathryn A, Ostrenga Joshua S, Rosen Bradley H, Hergenroeder Georgene, Bendy Lisa, Zhang Connie, Luo Nell Meosky, Ren Clement L
Аннотация
RATIONALE: Given improvement in symptoms and lung function in people with cystic fibrosis (CF) taking elexacaftor/tezacaftor/ivacaftor (ETI), there is increasing interest in de-escalation of care although the effect on outcomes remains unknown.
OBJECTIVES: To determine if self-reported discontinuation of chronic daily therapies (CDT) is associated with changes in pulmonary exacerbations and lung function in individuals taking ETI.
METHODS: The Home Reported Outcomes in Cystic Fibrosis 2 study was a 12-month prospective observational study in individuals age 12 and older taking ETI. This remote study collected patient-reported use and prior discontinuation of CDT at study entry via the Folia Health application. Primary outcomes were lung function and intravenous-antibiotic treated pulmonary exacerbations (PEx) as reported in the CF Foundation Patient Registry. Inverse probability weighting was used to compare outcomes between those that did and did not discontinue a CDT.
RESULTS: A total of 860 individuals consented, and 709 were included in these analyses. At baseline, 41.5% self-reported discontinuation of 1 or more CDTs. Over 12 months, those who discontinued CDT did not have any significant difference in the probability of PEx (mean difference -2.0%, 95% CI -6.7, 2.8%) or rate of change in lung function (mean difference 0.41%, 95% CI -1.40, 2.22).
CONCLUSION: In individuals taking ETI, self-reported discontinuation of CDT was not associated with an increased risk of PEx or decline in lung function.
OBJECTIVES: To determine if self-reported discontinuation of chronic daily therapies (CDT) is associated with changes in pulmonary exacerbations and lung function in individuals taking ETI.
METHODS: The Home Reported Outcomes in Cystic Fibrosis 2 study was a 12-month prospective observational study in individuals age 12 and older taking ETI. This remote study collected patient-reported use and prior discontinuation of CDT at study entry via the Folia Health application. Primary outcomes were lung function and intravenous-antibiotic treated pulmonary exacerbations (PEx) as reported in the CF Foundation Patient Registry. Inverse probability weighting was used to compare outcomes between those that did and did not discontinue a CDT.
RESULTS: A total of 860 individuals consented, and 709 were included in these analyses. At baseline, 41.5% self-reported discontinuation of 1 or more CDTs. Over 12 months, those who discontinued CDT did not have any significant difference in the probability of PEx (mean difference -2.0%, 95% CI -6.7, 2.8%) or rate of change in lung function (mean difference 0.41%, 95% CI -1.40, 2.22).
CONCLUSION: In individuals taking ETI, self-reported discontinuation of CDT was not associated with an increased risk of PEx or decline in lung function.