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The measurement properties of tests and tools used in cystic fibrosis studies: a systematic review

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.

Time to get serious about the detection and monitoring of early lung disease in cystic fibrosis

Structural and functional defects within the lungs of children with cystic fibrosis (CF) are detectable soon after birth and progress throughout preschool years often without overt clinical signs or symptoms. By school age, most children have structural changes such as bronchiectasis or gas trapping/hypoperfusion and lung function abnormalities that persist into later life. Despite improved survival, gains in forced expiratory volume in one second (FEV1) achieved across successive birth cohorts during childhood have plateaued, and rates of FEV1 decline in adolescence and adulthood have not slowed. This suggests that interventions aimed at preventing lung disease should be targeted to mild disease and commence in early life.

Role of Tris-CaEDTA as an adjuvant with nebulised tobramycin in cystic fibrosis patients with Pseudomonas aeruginosa lung infections: A randomised controlled trial

We tested if disrupting iron utilisation by P. aeruginosa by adding the Tris-buffered chelating agent CaEDTA to nebulised tobramycin would enhance bacterial clearance and improve lung function in CF patients.

Trial refresh: A case for an adaptive platform trial for pulmonary exacerbations of cystic fibrosis

This review will discuss the necessary steps required for a Bayesian adaptive platform trial to optimize treatment of pulmonary exacerbations of cystic fibrosis

Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized Medicine

In this Review, we consolidate the latest updates in the development of primary epithelial cellular model systems relevant for cystic fibrosis

Interleukin-1 is associated with inflammation and structural lung disease in young children with cystic fibrosis

Our data associates IL-1α with early structural lung damage in CF and suggests this pathway as a novel anti-inflammatory target

Use of a primary epithelial cell screening tool to investigate phage therapy in cystic fibrosis

This study demonstrates the feasibility of utilizing pre-clinical in vitro culture models to screen therapeutic candidates

CrossTalk opposing view: Mucosal acidification does not drive early progressive lung disease in cystic fibrosis

Whether airway mucosal acidification drives early progressive lung disease is controversial

The association between Staphylococcus aureus and subsequent bronchiectasis in children with cystic fibrosis

De novo S. aureus acquisition at age 3 is associated with later bronchiectasis and FEF25-75 in children with CF

Effect of posture on lung ventilation distribution and associations with structure in children with cystic fibrosis

Background: We assessed the effect of posture on ventilation distribution and the impact on associations with structural lung disease.