Effectivenesss of ivacaftor in severe cystic fibrosis patients and non‐G551D gating mutations

Background Ivacaftor is a significant innovation in the treatment of cystic fibrosis (CF) with gating mutations. A substantial percentage of patients with CF have severe lung involvement, but these patients are usually excluded from phase III clinical trials. Thus, the effectiveness of ivacaftor in...

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Bibliographic Details
Published in:Pediatric pulmonology 2019-09, Vol.54 (9), p.1398-1403
Main Authors: Salvatore, Donatello, Carnovale, Vincenzo, Iacotucci, Paola, Braggion, Cesare, Castellani, Carlo, Cimino, Giuseppe, Colangelo, Carmela, Francalanci, Michela, Leonetti, Giuseppina, Lucidi, Vincenzina, Manca, Antonio, Vitullo, Pamela, Ferrara, Nicola
Format: Article
Language:English
Subjects:
Adolescent
Adult
Aminophenols - therapeutic use
CFTR
Child
Chloride Channel Agonists - therapeutic use
Chlorides - analysis
Compassionate Use Trials
Cystic fibrosis
Cystic Fibrosis - drug therapy
Cystic Fibrosis - genetics
Cystic Fibrosis Transmembrane Conductance Regulator - genetics
Female
Forced Expiratory Volume
gating mutations
Humans
Ion Channel Gating - genetics
Ivacaftor
Lung - physiopathology
Male
Middle Aged
Mutation
Pediatrics
Quinolones - therapeutic use
Respiratory Function Tests
Respiratory therapy
Retrospective Studies
Sweat - chemistry
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Summary:Background Ivacaftor is a significant innovation in the treatment of cystic fibrosis (CF) with gating mutations. A substantial percentage of patients with CF have severe lung involvement, but these patients are usually excluded from phase III clinical trials. Thus, the effectiveness of ivacaftor in this population has not been fully determined. Methods Data were collected from Italian CF centers with patients enrolled in an ivacaftor compassionate use programme (percent predicted [pp] forced expiratory volume in 1 second [FEV1] 
ISSN:8755-6863
1099-0496
DOI:10.1002/ppul.24424