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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.

Overcoming Challenges to Make Bacteriophage Therapy Standard Clinical Treatment Practice for Cystic Fibrosis

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.

Minimal structural lung disease in early life represents significant pathology

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.

Structural determinants of long term functional outcomes in young children with cystic fibrosis

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

Early lung disease in infants and preschool children with cystic fibrosis: What have we learned and what should we do about it?

This review summarizes what we have learned about early lung disease in children with CF and discusses the implications for clinical practice and research

Chest imaging in cystic fibrosis studies: What counts, and can be counted?

The aim of this study is to characterize the role of currently available CT and MRI markers in clinical studies, and to discuss challenges with CF studies.

The effect of 100% oxygen on tidal breathing parameters in preschool children

This study aimed to determine whether breathing 100% oxygen altered tidal breathing parameters in healthy young children and young children with CF.

Hypoxia and sterile inflammation in cystic fibrosis airways: Mechanisms and potential therapies

In this review, we consider recent evidence regarding hypoxia and sterile inflammation in cystic fibrosis airways