Calibration and validation of modeled 5-year survival predictions among people with cystic fibrosis treated with the cystic fibrosis transmembrane conductance regulator modulator ivacaftor using United States registry data

OBJECTIVES: Cystic fibrosis (CF) is a rare genetic disease characterized by life-shortening lung function decline. Ivacaftor, a CF transmembrane conductance regulator modulator (CFTRm), was approved in 2012 for people with CF with specific gene mutations. We used real-world evidence of 5-year mortal...

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Detalles Bibliográficos
Autores principales: McGarry, Lisa J., Bhaiwala, Zahra, Lopez, Andrea, Chandler, Conor, Pelligra, Christopher G., Rubin, Jaime L., Liou, Theodore G.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2023
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10096446/
https://www.ncbi.nlm.nih.gov/pubmed/37043485
http://dx.doi.org/10.1371/journal.pone.0283479

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10096446/
https://www.ncbi.nlm.nih.gov/pubmed/37043485
http://dx.doi.org/10.1371/journal.pone.0283479

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