Evidence map›Paper›PMID 31409396›Full record

Observational studyBMC pulmonary medicine2019

Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional study.

Scott C Bell, Jochen G Mainz, Gordon MacGregor, Susan Madge, Julie Macey, Moshe Fridman, Ellison D Suthoff, Siva Narayanan, Nils Kinnman

Abstract readMulticenter StudyObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in BMC pulmonary medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed, 3 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

10 citing papers in PubMed, 3 syntheses or guidelines pooled it.

  1. Pooled it
  2. Use of elexacaftor+tezacaftor+ivacaftor in individuals with cystic fibrosis and at least one F508del allele: a systematic review and meta-analysis.Jornal brasileiro de pneumologia : publicacao oficial da Sociedade Brasileira de Pneumologia e Tisilogia · 2024
    Pooled it
  3. The measurement properties of tests and tools used in cystic fibrosis studies: a systematic review.European respiratory review : an official journal of the European Respiratory Society · 2021
    Pooled it
  4. Article
  5. Article
  6. Employment Status and Work Ability in Adults with Cystic Fibrosis.International journal of environmental research and public health · 2021
    Article
  7. Article
  8. Review
  9. Long term clinical effectiveness of ivacaftor in people with the G551D CFTR mutation.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2021
    Observational
  10. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Scott C BellDepartment of Thoracic Medicine, The Prince Charles Hospital and QIMR Berghofer Medical Research Institute, Brisbane, QLD, Australia. scott.bell@qimrberghofer.edu.au.
Jochen G MainzJena University Hospital, Jena, Germany.
Gordon MacGregorGartnavel General Hospital, Glasgow, UK.
Susan MadgeRoyal Brompton Hospital, London, UK.
Julie MaceyUniversity Hospital Bordeaux, Bordeaux, France.
Moshe FridmanAMF Consulting, Los Angeles, CA, USA.
Ellison D SuthoffFormerly of Vertex Pharmaceuticals Incorporated, Boston, MA, USA.
Siva NarayananDecision Resources Group, Burlington, MA, USA.
Nils KinnmanVertex Pharmaceuticals Incorporated, Boston, MA, USA.

Funding

Vertex Pharmaceuticals Incorporated NA
6 · The paper itself

Abstract

backgroundClinical 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.

methodsPatients 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.

resultsAfter 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.

conclusionsG551D/IVA patients reported better HRQoL than F508del/SOC patients on generic and disease-specific measures in a real-world setting.

Indexed as

AminophenolsAminopyridinesBenzodioxolesChildCross-Sectional StudiesCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDrug CombinationsFemaleForced Expiratory VolumeHumansInternationalityMaleMultivariate AnalysisMutationPatient Reported Outcome MeasuresAminophenolsAminopyridinesBenzodioxolesCFTR protein, humanCystic Fibrosis Transmembrane Conductance Regulatorcystic fibrosis transmembrane conductance regulator delta F508Drug Combinationslumacaftor, ivacaftor drug combinationQuinolonesCystic fibrosisG551D-CFTRIvacaftorPatient-reported outcomesWork productivity

Identifiers

PMID31409396
PMCPMC6693259

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.