Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional study.
Aminophenols
/ therapeutic use
Aminopyridines
/ therapeutic use
Benzodioxoles
/ therapeutic use
Child
Cross-Sectional Studies
Cystic Fibrosis
/ drug therapy
Cystic Fibrosis Transmembrane Conductance Regulator
/ genetics
Drug Combinations
Female
Forced Expiratory Volume
Humans
Internationality
Male
Multivariate Analysis
Mutation
Patient Reported Outcome Measures
Quality of Life
Quinolones
/ therapeutic use
Regression Analysis
Surveys and Questionnaires
Cystic fibrosis
G551D-CFTR
Ivacaftor
Patient-reported outcomes
Work productivity
Journal
BMC pulmonary medicine
ISSN: 1471-2466
Titre abrégé: BMC Pulm Med
Pays: England
ID NLM: 100968563
Informations de publication
Date de publication:
13 Aug 2019
13 Aug 2019
Historique:
received:
18
03
2019
accepted:
26
06
2019
entrez:
15
8
2019
pubmed:
15
8
2019
medline:
31
1
2020
Statut:
epublish
Résumé
Clinical studies demonstrate that ivacaftor (IVA) improves health-related quality of life (HRQoL) in patients aged ≥6 years with cystic fibrosis (CF). The real-world impact of IVA and standard of care (SOC) in groups of patients with G551D and F508del mutations, respectively, was assessed using a survey comprising disease-specific and generic HRQoL measures. Patients with CF aged ≥12 years, or aged 6-11 years with caregiver support, with either (1) a G551D mutation and receiving IVA (G551D/IVA) for ≥3 months, or (2) homozygous for F508del and receiving SOC before lumacaftor/IVA availability (F508del/SOC), were eligible to participate in a cross-sectional survey. Demographic and clinical characteristics, and HRQoL measures were compared between patient groups, and multiple regression analyses were conducted. After differences in patient demographic and clinical characteristics were controlled for, significantly better scores were observed in the G551D/IVA group than in the F508del/SOC group on multiple domains of the validated Cystic Fibrosis Questionnaire-Revised and the EuroQol 5-dimensions 5-level questionnaire. G551D/IVA patients reported better HRQoL than F508del/SOC patients on generic and disease-specific measures in a real-world setting.
Sections du résumé
BACKGROUND
BACKGROUND
Clinical studies demonstrate that ivacaftor (IVA) improves health-related quality of life (HRQoL) in patients aged ≥6 years with cystic fibrosis (CF). The real-world impact of IVA and standard of care (SOC) in groups of patients with G551D and F508del mutations, respectively, was assessed using a survey comprising disease-specific and generic HRQoL measures.
METHODS
METHODS
Patients with CF aged ≥12 years, or aged 6-11 years with caregiver support, with either (1) a G551D mutation and receiving IVA (G551D/IVA) for ≥3 months, or (2) homozygous for F508del and receiving SOC before lumacaftor/IVA availability (F508del/SOC), were eligible to participate in a cross-sectional survey. Demographic and clinical characteristics, and HRQoL measures were compared between patient groups, and multiple regression analyses were conducted.
RESULTS
RESULTS
After differences in patient demographic and clinical characteristics were controlled for, significantly better scores were observed in the G551D/IVA group than in the F508del/SOC group on multiple domains of the validated Cystic Fibrosis Questionnaire-Revised and the EuroQol 5-dimensions 5-level questionnaire.
CONCLUSIONS
CONCLUSIONS
G551D/IVA patients reported better HRQoL than F508del/SOC patients on generic and disease-specific measures in a real-world setting.
Identifiants
pubmed: 31409396
doi: 10.1186/s12890-019-0887-6
pii: 10.1186/s12890-019-0887-6
pmc: PMC6693259
doi:
Substances chimiques
Aminophenols
0
Aminopyridines
0
Benzodioxoles
0
CFTR protein, human
0
Drug Combinations
0
Quinolones
0
cystic fibrosis transmembrane conductance regulator delta F508
0
lumacaftor, ivacaftor drug combination
0
Cystic Fibrosis Transmembrane Conductance Regulator
126880-72-6
Types de publication
Journal Article
Multicenter Study
Observational Study
Langues
eng
Sous-ensembles de citation
IM
Pagination
146Subventions
Organisme : Vertex Pharmaceuticals Incorporated
ID : NA
Références
Ann Med. 2001 Jul;33(5):337-43
pubmed: 11491192
Lancet. 2003 May 17;361(9370):1671-6
pubmed: 12767731
Am J Hum Genet. 1992 Aug;51(2):245-50
pubmed: 1379413
J Pediatr Psychol. 2003 Dec;28(8):535-45
pubmed: 14602844
Chest. 2005 Oct;128(4):2347-54
pubmed: 16236893
Proc Am Thorac Soc. 2007 Aug 1;4(4):370-7
pubmed: 17652504
Chest. 2009 Jun;135(6):1610-1618
pubmed: 19447923
BMC Pediatr. 2009 Aug 28;9:55
pubmed: 19715563
QJM. 2009 Nov;102(11):793-8
pubmed: 19734299
Lancet. 2010 Mar 20;375(9719):1007-13
pubmed: 20304245
Pediatr Pulmonol. 2011 Jan;46(1):36-44
pubmed: 20848580
Chest. 2011 Dec;140(6):1598-1603
pubmed: 21659431
N Engl J Med. 2011 Nov 3;365(18):1663-72
pubmed: 22047557
Am J Respir Crit Care Med. 2013 Jun 1;187(11):1219-25
pubmed: 23590265
Am J Respir Crit Care Med. 2014 Jul 15;190(2):175-84
pubmed: 24927234
Thorax. 2014 Dec;69(12):1090-7
pubmed: 25246663
Lancet Respir Med. 2014 Nov;2(11):902-910
pubmed: 25311995
Genet Med. 2016 Apr;18(4):333-40
pubmed: 26087176
Health Qual Life Outcomes. 2015 Jul 02;13:93
pubmed: 26135562
Pediatr Pulmonol. 2015 Oct;50 Suppl 40:S74-9
pubmed: 26335957
BMC Health Serv Res. 2015 Sep 28;15:428
pubmed: 26416027
Thorax. 2016 Jan;71(1):26-34
pubmed: 26452630
Thorax. 2016 Jun;71(6):493-500
pubmed: 27030578
Health Qual Life Outcomes. 2016 Apr 21;14:63
pubmed: 27097977
Lancet. 2016 Nov 19;388(10059):2519-2531
pubmed: 27140670
J Pain Symptom Manage. 2016 Nov;52(5):681-687
pubmed: 27693896
Appl Health Econ Health Policy. 2017 Apr;15(2):127-137
pubmed: 28194657
Am J Respir Crit Care Med. 2017 Jun 15;195(12):1673-1676
pubmed: 28617084