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Research

A standardised framework for selecting outcomes for evaluation in trials has been proposed by the Core Outcome Measures in Effectiveness Trials working group. However, this method does not specify how to ensure that the outcomes that are selected are causally related to the disease and the health intervention being studied. Causal network diagrams may help researchers identify outcomes that are both clinically meaningful and likely to be causally dependent on the intervention, and endpoints that are, in turn, causally dependent on those outcomes.

Research

There is no consensus on how best to measure responses to interventions among children and adults with cystic fibrosis (CF). We have systematically reviewed and summarised the characteristics and measurement properties of tests and tools that have been used to capture outcomes in studies among people with CF, including their reliability, validity and responsiveness. This review is intended to guide researchers when selecting tests or tools for measuring treatment effects in CF trials. A consensus set of these tests and tools could improve consistency in how outcomes are captured and thereby facilitate comparisons and synthesis of evidence across studies.

Research

Individuals with cystic fibrosis (CF) are given antimicrobials as prophylaxis against bacterial lung infection, which contributes to the growing emergence of multidrug resistant (MDR) pathogens isolated. Pathogens such as Pseudomonas aeruginosa that are commonly isolated from individuals with CF are armed with an arsenal of protective and virulence mechanisms, complicating eradication and treatment strategies.

Research

Neutrophil elastase is a significant risk factor for structural lung disease in cystic fibrosis, and Pseudomonas aeruginosa airway infection is linked with neutrophilic inflammation and substantial respiratory morbidity. We aimed to evaluate how neutrophil elastase (NE) activity changes after P. aeruginosa eradication and influences early disease outcomes. We assessed participants in the AREST CF cohort between 2000 and 2018 who had P. aeruginosa cultured from their routine annual bronchoalveolar lavage (BAL) fluid and who underwent eradication treatment and a post eradication BAL. Factors associated with persistent P. aeruginosa infection, persistent neutrophilic inflammation following eradication and worse structural lung disease one year post-eradication were evaluated.

Dissecting the regulation of bile-induced biofilm formation in Staphylococcus aureus. Aspiration of bile into the cystic fibrosis (CF) lung has

Research

In this cohort, as compared with the AREST CF cohort, the authors highlight the limited correlation between infection and inflammation with lung function and structural impairment, and that this was mainly explained by the mild changes identified in lung function and on chest CT scan.

Parental Experience of Information and Education Processes Following Diagnosis of Their Infant With Cystic Fibrosis Via Newborn Screening. Abstract

Research

Chest CT identifies children at an early age who have adverse long-term outcomes

Lung Clearance Index and Structural Lung Disease on Computed Tomography in Early Cystic Fibrosis Abstract Rationale: The lung clearance index is a

Research

Here we examine the latest findings of neutrophils in pediatric CF lung disease and proposed mechanisms of their pathogenicity

Research

Aspergillus species and P. aeruginosa are commonly present in the lower airways from infancy

Quantitative assessment of airway dimensions in youngchildren with cystic fibrosis lung disease using chestcomputed tomography Abstract Objective: To

Quantification of CT bronchiectasis and its relationship to ventilation in cystic fibrosis Editorial by Tim Rosenow

The clinical utility of lung clearance index in early cystic fibrosis lung disease is not impacted by the number of multiple-breath washout trials

The clinical significance of oropharyngeal cultures in young children with cystic fibrosis ABSTRACT In children with cystic fibrosis (CF) the

Initial acquisition and succession of the cystic fibrosis lung microbiome is associated with disease progression in infants and preschool children

Assessment of p.Phe508del-CFTR functional restoration in pediatric primary cystic fibrosis airway epithelial cells Abstract Background Mutations in

The potential of antisense oligonucleotide therapies for inherited childhood lung diseases Antisense oligonucleotides (AOs) are an emerging