Trial reportHealth and quality of life outcomes2016
Impact of pulmonary exacerbations and lung function on generic health-related quality of life in patients with cystic fibrosis.
Trial report in Health and quality of life outcomes, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00909532 (A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX-770 in Subjects With Cystic Fibrosis and the G551D Mutation), which is not on this map. Cited by 23 papers, 4 of them syntheses that pooled 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.
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.
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX-770 in Subjects With Cystic Fibrosis and the G551D Mutation
Who cites it
23 citing papers in PubMed, 4 syntheses or guidelines pooled it, 67 citations in OpenAlex.
- Ivacaftor-tezacaftor-elexacaftor, tezacaftor-ivacaftor and lumacaftor-ivacaftor for treating cystic fibrosis: a systematic review and economic evaluation.Health technology assessment (Winchester, England) · 2025Pooled it
- Tools used to measure quality of life in adults with cystic fibrosis- a systematic review.Health and quality of life outcomes · 2025Pooled it
- Pooled it
- Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis.The Cochrane database of systematic reviews · 2019Pooled it
- The Impact of Activated Phosphoinositide 3-Kinase δ Syndrome (APDS) on Health-Related Quality of Life (HRQoL): Elicitation of Health State Utility Values Through Time Trade-Off (TTO) and EQ-5D.Advances in therapy · 2025Article
- Interrelationships between diet quality and health-related quality of life in Irish adults living with cystic fibrosis.European journal of nutrition · 2025Article
- Allergic Bronchopulmonary Aspergillosis (ABPA) in the Era of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators.Journal of fungi (Basel, Switzerland) · 2024Review
- Appropriateness of the EQ-5D-5L in capturing health-related quality of life in individuals with transfusion-dependent β-thalassemia: a mixed methods study.Health and quality of life outcomes · 2024Article
- Should atelectasis be considered a pulmonary complication and indicator of poor prognosis in cystic fibrosis?The Journal of international medical research · 2024Observational
- Psychometric performance of the CFQ-R-8D compared to the EQ-5D-3L and SF-6D in people with cystic fibrosis.Journal of patient-reported outcomes · 2024Article
- Article
- The estimation of health state utility values in rare diseases: do the approaches in submissions for NICE technology appraisals reflect the existing literature? A scoping review.The European journal of health economics : HEPAC : health economics in prevention and care · 2023Article
- Diet quality in cystic fibrosis - associations with patient reported outcome measures and enablers and barriers to eating a healthy diet: A protocol paper for a mixed methods study.HRB open research · 2022Article
- Cost-effectiveness of implementing routine hearing screening using a tablet audiometer for pediatric cystic fibrosis patients receiving high-dose IV aminoglycosides.Journal of managed care & specialty pharmacy · 2021Article
- Genetic Variation Near chrXq22-q23 Is Linked to Emotional Functioning in Cystic Fibrosis.Biological research for nursing · 2020Article
- Health State Utility Data in Cystic Fibrosis: A Systematic Review.PharmacoEconomics - open · 2020Review
- The future of cystic fibrosis care: a global perspective.The Lancet. Respiratory medicine · 2020Review
- Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional study.BMC pulmonary medicine · 2019Observational
- CyFiT telehealth: protocol for a randomised controlled trial of an online outpatient physiotherapy service for children with cystic fibrosis.BMC pulmonary medicine · 2019Article
- CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine.Frontiers in pharmacology · 2019Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThe analysis aimed to examine the impact of pulmonary exacerbations (PEs) and lung function on generic measures of HRQL in patients with cystic fibrosis (CF) using trial-based data.
methodsIn a 48-week randomized, placebo-controlled study of ivacaftor in patients ≥12 years with CF and a G551D-CFTR mutation the relationship between PEs, PE-related hospitalizations and percent predicted forced expiratory volume in one second (ppFEV1) with EQ-5D measures (index and visual analog scale [VAS]) was examined in post-hoc analyses. Multivariate mixed-effects models were employed to describe the association of PEs, PE-related hospitalizations, and ppFEV1 on EQ-5D measures.
resultsOne hundred sixty one patients (age: mean 25.5 [SD 9.5] years; baseline ppFEV1: 63.6 [16.4]) contributed 1,214 observations (ppFEV1: no lung dysfunction [n = 157], mild [n = 419], moderate [n = 572], severe [n = 66]). Problems were most frequently reported on pain/discomfort, anxiety/depression, and usual activities EQ-5D items. The mean (SE) EQ-5D index nominally decreased (worsened) with worsening severity of lung dysfunction (P = 0.070): 0.931 (0.023); mild: 0.923 (0.021); moderate: 0.904 (0.018); severe: 0.870 (0.020). 146 PEs were experienced by 72 patients, including 52 PEs (35.6 %) that required hospitalization. Mean EQ-5D index and VAS scores were lowest (worst) within 1 week (before or after PE start) for PEs requiring hospitalization. Pulmonary exacerbations, PE-related hospitalizations, and ppFEV1 were significant predictors of EQ-5D index and VAS.
conclusionsIn a clinical study of patients with CF (≥12 years of age and a G551D-CFTR mutation), PEs, primarily those requiring hospitalization, were associated with low EQ-5D index and VAS scores. The impact of ppFEV1 was relatively smaller. Reducing PEs, in particular those requiring hospitalization, would likely improve HRQL among these patients.
trial registrationClinicalTrials.gov, NCT00909532 ; URL: clinicaltrials.gov, May 26, 2009.
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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.