Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional study
Abstract 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...
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| Format: | Article |
| Language: | English |
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BMC
2019-08-01
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| Series: | BMC Pulmonary Medicine |
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| Online Access: | http://link.springer.com/article/10.1186/s12890-019-0887-6 |
| _version_ | 1818242574427619328 |
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| author | Scott C. Bell Jochen G. Mainz Gordon MacGregor Susan Madge Julie Macey Moshe Fridman Ellison D. Suthoff Siva Narayanan Nils Kinnman |
| author_facet | Scott C. Bell Jochen G. Mainz Gordon MacGregor Susan Madge Julie Macey Moshe Fridman Ellison D. Suthoff Siva Narayanan Nils Kinnman |
| author_sort | Scott C. Bell |
| collection | DOAJ |
| description | Abstract 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 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 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 G551D/IVA patients reported better HRQoL than F508del/SOC patients on generic and disease-specific measures in a real-world setting. |
| first_indexed | 2024-12-12T13:47:24Z |
| format | Article |
| id | doaj.art-4efd007193ce4914a086b5bfedab51a4 |
| institution | Directory Open Access Journal |
| issn | 1471-2466 |
| language | English |
| last_indexed | 2024-12-12T13:47:24Z |
| publishDate | 2019-08-01 |
| publisher | BMC |
| record_format | Article |
| series | BMC Pulmonary Medicine |
| spelling | doaj.art-4efd007193ce4914a086b5bfedab51a42022-12-22T00:22:38ZengBMCBMC Pulmonary Medicine1471-24662019-08-011911910.1186/s12890-019-0887-6Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional studyScott C. Bell0Jochen G. Mainz1Gordon MacGregor2Susan Madge3Julie Macey4Moshe Fridman5Ellison D. Suthoff6Siva Narayanan7Nils Kinnman8Department of Thoracic Medicine, The Prince Charles Hospital and QIMR Berghofer Medical Research InstituteJena University HospitalGartnavel General HospitalRoyal Brompton HospitalUniversity Hospital BordeauxAMF ConsultingFormerly of Vertex Pharmaceuticals IncorporatedDecision Resources GroupVertex Pharmaceuticals IncorporatedAbstract 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 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 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 G551D/IVA patients reported better HRQoL than F508del/SOC patients on generic and disease-specific measures in a real-world setting.http://link.springer.com/article/10.1186/s12890-019-0887-6Patient-reported outcomesWork productivityIvacaftorCystic fibrosisG551D-CFTR |
| spellingShingle | Scott C. Bell Jochen G. Mainz Gordon MacGregor Susan Madge Julie Macey Moshe Fridman Ellison D. Suthoff Siva Narayanan Nils Kinnman Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional study BMC Pulmonary Medicine Patient-reported outcomes Work productivity Ivacaftor Cystic fibrosis G551D-CFTR |
| title | Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional study |
| title_full | Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional study |
| title_fullStr | Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional study |
| title_full_unstemmed | Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional study |
| title_short | Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional study |
| title_sort | patient reported outcomes in patients with cystic fibrosis with a g551d mutation on ivacaftor treatment results from a cross sectional study |
| topic | Patient-reported outcomes Work productivity Ivacaftor Cystic fibrosis G551D-CFTR |
| url | http://link.springer.com/article/10.1186/s12890-019-0887-6 |
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