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Artikel: Real-world effectiveness of elexacaftor/tezacaftor/ivacaftor on the burden of illness in adolescents and adults with cystic fibrosis.

Keens, Thomas / Hoffman, Veena / Topuria, Ia / Elder, Ken / Cerf, Shannon / Mulder, Kyra / Roberts, Jon / Lysinger, Jerimiah / Del Carmen Reyes, Maria / Berdella, Maria / Cairns, Anne Marie / Jain, Manu / Ganapathy, Vaidyanathan / Lou, Yiyue / Morcos, Bassem / Wu, Chuntao / Sass, Laura

Heliyon

2024  Band 10, Heft 7, Seite(n) e28508

Abstract: Background: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to be safe and efficacious in people with cystic fibrosis (CF) aged ≥2 years. Here, we describe results from an observational study assessing change in burden of illness following ...

Abstract Background: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to be safe and efficacious in people with cystic fibrosis (CF) aged ≥2 years. Here, we describe results from an observational study assessing change in burden of illness following initiating ELX/TEZ/IVA in real-world settings.
Methods: This US-based, multicenter, observational study used data from electronic medical records to evaluate real-world burden of illness before and after ELX/TEZ/IVA initiation in people with CF aged ≥12 years heterozygous for
Results: Overall, 206 people with CF were enrolled (mean [SD] age 22.5 [11.1] years; 192 [93.2%] with
Conclusions: ELX/TEZ/IVA treatment was associated with improved lung function, increased BMI, reduced frequency of PEx, and improved (i.e., reduced) HbA1c. These results confirm the broad clinical benefits of ELX/TEZ/IVA seen in clinical trials and show the potential for ELX/TEZ/IVA to improve markers of glucose metabolism.
Sprache Englisch
Erscheinungsdatum 2024-03-26
Erscheinungsland England
Dokumenttyp Journal Article
ZDB-ID 2835763-2
ISSN 2405-8440
ISSN 2405-8440
DOI 10.1016/j.heliyon.2024.e28508
Datenquelle MEDical Literature Analysis and Retrieval System OnLINE

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