Real-World Long-Term Ivacaftor for Cystic Fibrosis in France: Clinical Effectiveness and Healthcare Resource Utilization.
Adults
Children
Cystic fibrosis
Exacerbation
Ivacaftor
Lung function
Real-world experience
Journal
Pulmonary therapy
ISSN: 2364-1746
Titre abrégé: Pulm Ther
Pays: United States
ID NLM: 101687144
Informations de publication
Date de publication:
Dec 2021
Dec 2021
Historique:
received:
08
03
2021
accepted:
22
04
2021
pubmed:
9
6
2021
medline:
9
6
2021
entrez:
8
6
2021
Statut:
ppublish
Résumé
Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has demonstrated clinical benefits in phase 3 trials. We report results from a real-world study (BRIO) to assess the effectiveness of ivacaftor in people with cystic fibrosis (pwCF) in France. BRIO was an observational study conducted at 35 centers in France. Both pwCF initiating ivacaftor treatment and those already taking ivacaftor were included and prospectively followed for 24 months. The primary objective was to evaluate the effect of ivacaftor on percent predicted forced expiratory volume in 1 s (ppFEV A total of 129 pwCF were enrolled; 58.9% were aged < 18 years; 64.3% had a G551D-CFTR allele. Mean age at ivacaftor initiation was 19.1 years (range, 2-64 years); ppFEV The results from this real-world study of ivacaftor usage in France were consistent with prior clinical trial outcomes, confirming the clinical effectiveness of ivacaftor, as well as an associated reduction in HCRU.
Identifiants
pubmed: 34101145
doi: 10.1007/s41030-021-00158-5
pii: 10.1007/s41030-021-00158-5
pmc: PMC8589905
doi:
Types de publication
Journal Article
Langues
eng
Pagination
455-468Investigateurs
Sophie Ramel
(S)
Philippe Vigneron
(P)
Veronique Storni
(V)
Natacha Remus
(N)
Laurence Bassinet Isabelle Durieu
(LBI)
Muriel Laurans
(M)
Cinthia Rames Françoise Troussier
(CRF)
Marie-Laure Dalphin Frédéric Huet
(MDF)
Isabelle Pin
(I)
Boubou Camara Annabelle Payet
(BCA)
Jeanne Languepin
(J)
Isabelle Danner-Boucher
(I)
Marc Albertini Julie Mankikian
(MAJ)
Nathalie Wizla
(N)
Laure Cosson
(L)
Eric Deneuville Marie Mittaine
(EDM)
Philippe Reix
(P)
Nadine Dufeu
(N)
Valérie David
(V)
Thierry Langin Stéphanie Bui
(TLS)
Raphael Chiron
(R)
Dominique Grenet
(D)
Frédérique Chedevergne
(F)
Chantal Belleguic
(C)
Laurent Mely
(L)
Commentaires et corrections
Type : ErratumIn
Informations de copyright
© 2021. The Author(s).
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