Profiling the response to lumacaftor-ivacaftor in children with cystic between fibrosis and new insight from a French-Italian real-life cohort.
Child
Humans
Adolescent
Cystic Fibrosis Transmembrane Conductance Regulator
/ therapeutic use
Aminophenols
/ therapeutic use
Benzodioxoles
/ therapeutic use
Aminopyridines
/ therapeutic use
Cystic Fibrosis
/ drug therapy
Forced Expiratory Volume
Drug Combinations
Anti-Bacterial Agents
/ therapeutic use
Fibrosis
Mutation
children
lumacaftor-ivacaftor
principal component analysis
Journal
Pediatric pulmonology
ISSN: 1099-0496
Titre abrégé: Pediatr Pulmonol
Pays: United States
ID NLM: 8510590
Informations de publication
Date de publication:
12 2022
12 2022
Historique:
revised:
11
08
2022
received:
22
04
2022
accepted:
21
08
2022
pubmed:
24
8
2022
medline:
22
11
2022
entrez:
23
8
2022
Statut:
ppublish
Résumé
Clinical trials for CFTR modulators consider mean changes of clinical status at the cohort level, and thus fail to assess the heterogeneity of the response. We aimed to study the different response profiles to lumacaftor-ivacaftor according to age in children with cystic fibrosis (CF). A mathematical framework, including principal component analysis, data clustering, and data completion, was applied to a multicenter cohort of 112 children aged 6-18 years, treated with lumacaftor-ivacaftor. Studied parameters at baseline and 6 months included body mass index (BMI), number of days of antibiotics (ATB), Sweat test (ST), forced expiratory volume in 1 s expressed in percentage predicted (ppFEV Change in ppFEV Change in ppFEV
Identifiants
pubmed: 35996214
doi: 10.1002/ppul.26123
pmc: PMC9826158
doi:
Substances chimiques
ivacaftor
1Y740ILL1Z
lumacaftor
EGP8L81APK
Cystic Fibrosis Transmembrane Conductance Regulator
126880-72-6
Aminophenols
0
Benzodioxoles
0
Aminopyridines
0
Drug Combinations
0
Anti-Bacterial Agents
0
Types de publication
Multicenter Study
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
2992-2999Informations de copyright
© 2022 The Authors. Pediatric Pulmonology published by Wiley Periodicals LLC.
Références
Am J Respir Crit Care Med. 2013 Jun 1;187(11):1219-25
pubmed: 23590265
N Engl J Med. 2015 Jul 16;373(3):220-31
pubmed: 25981758
Eur Respir J. 2022 May 5;59(5):
pubmed: 34675045
Chest. 2013 Jan;143(1):14-18
pubmed: 23276841
Lancet Respir Med. 2017 Jul;5(7):557-567
pubmed: 28606620
Am J Respir Crit Care Med. 2021 Jun 15;203(12):1522-1532
pubmed: 33734030
J Cyst Fibros. 2019 Sep;18(5):708-713
pubmed: 31253540
Genes (Basel). 2019 Feb 26;10(3):
pubmed: 30813645
J Pediatr. 1997 Dec;131(6):809-14
pubmed: 9427882
Ann Am Thorac Soc. 2021 Jan;18(1):75-83
pubmed: 32644818
Am J Respir Crit Care Med. 2020 Jan 15;201(2):188-197
pubmed: 31601120
J Cyst Fibros. 2016 Jul;15(4):416-23
pubmed: 27316663
Front Immunol. 2020 Jun 30;11:1198
pubmed: 32695100
Am J Respir Crit Care Med. 2018 Aug 15;198(4):526-528
pubmed: 29614238
Chest. 2002 Jan;121(1):64-72
pubmed: 11796433
Eur Respir J. 2012 Dec;40(6):1324-43
pubmed: 22743675
Thorax. 2014 Jun;69(6):586-7
pubmed: 24258833
N Engl J Med. 2017 Nov 23;377(21):2013-2023
pubmed: 29099344
Pediatr Pulmonol. 1990;8(1):16-22
pubmed: 2405342
Bioinformatics. 2012 Jan 1;28(1):112-8
pubmed: 22039212
Lancet Respir Med. 2018 Jul;6(7):483-484
pubmed: 29886025
Paediatr Respir Rev. 2008 Sep;9(3):176-80
pubmed: 18694709
J Cyst Fibros. 2019 May;18(3):368-374
pubmed: 30595473
Pediatr Pulmonol. 2022 Dec;57(12):2992-2999
pubmed: 35996214
Am J Respir Crit Care Med. 2017 Apr 1;195(7):912-920
pubmed: 27805836