AnnalsATS 2022: Rigor, Value, Impact

Vollständiger Abstract

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<h4>Rationale</h4>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.<h4>Objectives</h4>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.<h4>Methods</h4>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.<h4>Results</h4>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).<h4>Conclusion</h4>In individuals taking ETI, self-reported discontinuation of CDT was not associated with an increased risk of PEx or decline in lung function.

Abstract: PubMed · Datensatz