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Updated: Jan 1, 2026
Establishment of a Clinic-based Biorepository
Published on: May 29, 2017
Utilizing centralized biorepository samples for biomarkers of cystic fibrosis lung disease severity
Scott D Sagel1, Brandie D Wagner2, Assem Ziady3
1Department of Pediatrics, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO, USA.
Insights
Blood biomarkers can distinguish children with cystic fibrosis (CF) based on lung disease severity. Inflammatory proteins like C-reactive protein were higher in severe CF cases, aiding biomarker discovery.
Area of Science:
- Biomarker discovery
- Pediatric respiratory diseases
- Inflammation research
Background:
- Circulating biomarkers are crucial for managing cystic fibrosis (CF) lung disease and advancing drug development.
- Identifying blood-based markers can improve patient care and research in CF.
- This study investigated blood biomarkers to differentiate CF children by lung disease severity.
Purpose of the Study:
- To test the hypothesis that blood-based biomarkers can distinguish between CF children with mild versus severe lung disease.
- To identify specific inflammatory proteins, lipids, and metabolites associated with CF lung disease severity.
Main Methods:
- Serum samples from CF children, categorized as 'mild' or 'severe' based on lung function, were analyzed.
- Samples were matched for age, gender, CFTR genotype, and Pseudomonas aeruginosa infection status.
- Targeted assays measured inflammatory proteins, lipids, and metabolite profiles.
Main Results:
- Children with severe CF had significantly higher levels of C-reactive protein, serum amyloid A, granulocyte colony-stimulating factor, and calprotectin.
- The mild CF group showed higher serum linoleic acid concentrations.
- Lower arabitol levels were observed in the severe CF cohort, with other metabolic pathways showing no significant differences after adjustment.
Conclusions:
- Distinct circulating inflammatory proteins differentiate CF children based on lung function.
- High-quality, linked patient samples are valuable for biomarker development in CF research.
- These findings support the use of blood biomarkers for assessing CF lung disease severity.
Background:
Circulating biomarkers reflective of lung disease activity and severity have the potential to improve patient care and accelerate drug development in CF. The objective of this study was to leverage banked specimens to test the hypothesis that blood-based biomarkers discriminate CF children segregated by lung disease severity.
Methods:
Banked serum samples were selected from children who were categorized into two extremes of phenotype associated with lung function ('mild' or 'severe') based on CF-specific data and were matched on age, gender, CFTR genotype, and P. aeruginosa infection status. Targeted inflammatory proteins, lipids, and discovery metabolite profiles were measured in these serum samples.
Results:
The severe cohort, characterized by a lower CF-specific FEV1 percentile, had significantly higher circulating concentrations of high sensitivity C-reactive protein, serum amyloid A, granulocyte colony stimulating factor, and calprotectin compared to the mild cohort. The mild cohort tended to have higher serum linoleic acid concentrations. The metabolite arabitol was lower in the severe cohort while other CF relevant metabolic pathways showed non-significant differences after adjusting for multiple comparisons. A sensitivity analysis to correct for biased estimates that may result from selecting subjects using an extremes of phenotype approach confirmed the protein biomarker findings.
Conclusions:
Circulating inflammatory proteins differ in CF children segregated by lung function. These findings serve to demonstrate the value of maintaining centralized, high quality patient derived samples for future research, with linkage to clinical information to answer testable hypotheses in biomarker development.
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