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Pediatric pulmonology · 2025

Baseline Characteristics and Self-Reported Use of Chronic Daily Therapies of the Home Reported Outcomes With CFTR Modulator Therapy (HERO-2) Cohort.

СтатьяНаблюдательное исследованиеНазвание и аннотация: Перевод пока не подготовлен
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Hergenroeder Georgene E, Psoter Kevin J, Brown Cynthia D, Ostrenga Josh, Ford Christine, Bendy Lisa, Rosen Bradley H, Luo Nell Meosky, Zhang Connie, Sabadosa Kathryn A, Ren Clement L

Аннотация

BACKGROUND: The HERO-2 study is a prospective, observational study investigating chronic daily therapy (CDT) changes among people with cystic fibrosis (PwCF) ≥ 12 years of age taking elexacaftor/tezacaftor/ivacaftor (ETI). The goal of this analysis was to describe baseline characteristics and patterns of CDT discontinuation reported at study enrollment and identify clinical features associated with CDT discontinuation.

METHODS: Study participants were recruited through their CF center, community engagement, or a smartphone application (Folia Health). Study enrollment and participation took place remotely through Folia Health. Participants gave consent to link their data with the CF Foundation Patient Registry (CFFPR). At enrollment, participants completed a baseline survey describing CDT use since starting ETI. Descriptive statistics were used to summarize HERO-2 participant characteristics. Logistic regression evaluated the association of selected individual characteristics with odds of CDT discontinuation.

RESULTS: A total of 860 PwCF enrolled in HERO-2, and 755 (87.8%) completed the enrollment survey and were linked to the CFFPR. At enrollment, 313 participants (41.5%) reported discontinuation of at least one CDT. The most frequently discontinued CDT was airway clearance (22.4%). Intravenous (IV) antibiotic treatment for a pulmonary exacerbation in the prior year and public insurance were associated with lower odds of CDT discontinuation.

CONCLUSIONS: In this large cohort of PwCF taking ETI, approximately 42% reported discontinuation of at least one CDT. Providers should be aware of these CDT discontinuation patterns and engage in open dialogue with PwCF and their families about CDT de-escalation.

Предметные рубрики MeSH

ИП Урсолов Владлен Сергеевич

ИНН 500118482352 · ОГРНИП 314500117700049

Реквизиты и контакты