Radiological and long-term clinical response to elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with advanced lung disease.

Bente L Aalbers,Firdaus A A Mohamed Hoesein,Regina W Hofland,Inez Bronsveld,Marian A Kruijswijk,Suzan Schotman, Corlien W Slingerland, Hannah Panhuis,C Kors van der Ent,Harry G M Heijerman

Pediatric pulmonology（2023）

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摘要

pwCF with advanced lung disease experience relevant clinical benefit after 2 years of treatment with ELE/TEZ/IVA in a compassionate use setting. Structural lung damage, quality of life, exacerbation rate, and BMI improved significantly with treatment. Gain in ppFEV is lower compared to the phase III trials that included younger patients with moderately affected lung function.

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cystic fibrosis,advanced lung disease,elexacaftor/tezacaftor/ivacaftor,elexacaftor/tezacaftor/ivacaftor,elexacaftor/tezacaftor/ivacaftor

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