Evidence map›Paper›PMID 37568199›Full record

Observational studyRespiratory research2023

The clinical impact of Lumacaftor-Ivacaftor on structural lung disease and lung function in children aged 6-11 with cystic fibrosis in a real-world setting.

Paul McNally, Barry Linnane, Michael Williamson, Basil Elnazir, Christopher Short, Clare Saunders, Laura Kirwan, Rea David, Mariette P C Kemner-Van de Corput, Harm A W M Tiddens and 2 more

Open access · goldAbstract readObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in Respiratory research, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
3.4field-weighted citation impact, top 7% of its field
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

7 citing papers in PubMed, 13 citations in OpenAlex.

  1. Article
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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

12 authors at 7 institutions in 3 countries.

Paul McNallyRespiratory Department, Children's Health Ireland, Crumlin, Dublin, Ireland.
Barry LinnaneUniversity of Limerick School of Medicine, Limerick, Ireland.
Michael WilliamsonRespiratory Department, Children's Health Ireland, Crumlin, Dublin, Ireland.
Basil ElnazirRespiratory Department, Children's Health Ireland, Crumlin, Dublin, Ireland.
Christopher ShortNHLI, Imperial College, London, UK.
Clare SaundersNHLI, Imperial College, London, UK.
Laura KirwanCystic Fibrosis Registry of Ireland, Dublin, Ireland.
Rea DavidRespiratory Department, Children's Health Ireland, Crumlin, Dublin, Ireland.
Mariette P C Kemner-Van de CorputDepartment of Paediatric Pulmonology and Allergology, Department of Radiology and Nuclear Medicine, Erasmus Medical Centre - Sophia Children's Hospital, Rotterdam, The Netherlands.
Harm A W M TiddensRespiratory Department, Children's Health Ireland, Crumlin, Dublin, Ireland.
Jane C DaviesNHLI, Imperial College, London, UK.
Des W CoxRespiratory Department, Children's Health Ireland, Crumlin, Dublin, Ireland. desmond.cox@ucd.ie.
Children's Health Ireland at Crumlin · IEImperial College London · GBCystic Fibrosis Ireland · IEErasmus MC - Sophia Children’s Hospital · NLHarefield Hospital · GBUniversity College Dublin · IEUniversity of Limerick · IE

Funding

National Children's Research Centre Innovation grantVertex Pharmaceuticals IIS-2017-106550
6 · The paper itself

Abstract

backgroundData from clinical trials of lumacaftor-ivacaftor (LUM-IVA) demonstrate improvements in lung clearance index (LCI) but not in FEV

methodsThis real-world observational cohort study was conducted across four paediatric sites in Ireland over 24-months using spirometry-controlled CT scores and LCI as primary outcome measures. Children commencing LUM-/IVA as part of routine care were included. CT scans were manually scored with the PRAGMA CF scoring system and analysed using the automated bronchus-artery (BA) method. Secondary outcome measures included rate of change of ppFEV

resultsSeventy-one participants were recruited to the study, 31 of whom had spirometry-controlled CT performed at baseline, and after one year and two years of LUM/IVA treatment. At two years there was a reduction from baseline in trapped air scores (0.13 to 0.07, p = 0.016), but an increase from baseline in the % bronchiectasis score (0.84 to 1.23, p = 0.007). There was no change in overall % disease score (2.78 to 2.25, p = 0.138). Airway lumen to pulmonary artery ratios (A

conclusionIn a real-world setting, the use of LUM/IVA over two years in children with CF aged 6-11 resulted in improvements in air trapping on CT but worsening in bronchiectasis scores. Our results suggest that LUM/IVA use in this age group improves air trapping but does not prevent progression of bronchiectasis over two years of treatment.

Indexed as

BronchiectasisCystic FibrosisAminophenolsAminopyridinesBenzodioxolesChildCystic Fibrosis Transmembrane Conductance RegulatorDrug CombinationsHumansLungMutationQuinolonesAminophenolsAminopyridinesBenzodioxolesCystic Fibrosis Transmembrane Conductance RegulatorDrug CombinationsivacaftorlumacaftorQuinolonesChildrenLung clearance indexPRAGMA CF scoresReal-world studies

Identifiers

PMID37568199
PMCPMC10416528
OpenAlexW4385763208

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.