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Abstract
The cystic fibrosis (CF) community seeks to explain heterogeneous outcomes of pulmonary exacerbation (PEX) treatment. Serum and sputum inflammatory mediators may identify people with CF (PwCF) at risk for suboptimal responses. However, lack of an established association between response phenotypes and these mediators limits clinical application. In this pilot study, we prospectively characterized treatment response phenotypes by assessing health-related quality-of-life (HRQoL) during PEX. We also measured lung function and iron-related biochemical parameters in serum and sputum. We classified subjects as sustained symptom-responders (SRs) or non-sustained symptom-responders (NSRs) based on the absence or presence, respectively, of worsened symptom scores after initial improvement. We used linear mixed models (LMMs) to determine whether trends in lung function, hematologic, serum, and sputum indices of inflammation differed between response cohorts. In 20 PwCF, we identified 10 SRs and 10 NSRs with no significant differences in lung function at PEX onset and treatment durations. SRs had better model-predicted trends in lung function than NSRs during PEX. Non-linear trends in serum and sputum iron levels significantly differed between SRs and NSRs. In adults with cystic fibrosis, PEX treatment response phenotypes may be correlated with distinctive trends in serum and sputum iron concentrations.
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1 Dartmouth-Hitchcock Medical Center, Section of Pulmonary Medicine, 5C, Lebanon, USA (GRID:grid.413480.a) (ISNI:0000 0004 0440 749X)
2 University of Kansas Medical Center, Division of Pulmonary, Critical Care and Sleep Medicine, Kansas City, USA (GRID:grid.412016.0) (ISNI:0000 0001 2177 6375)
3 University of Kansas Medical Center, Biostatistics and Data Science, Kansas City, USA (GRID:grid.412016.0) (ISNI:0000 0001 2177 6375)
4 Maine Medical Center, Pulmonary and Critical Care Medicine, Portland, USA (GRID:grid.240160.1)
5 Geisel School of Medicine at Dartmouth, Microbiology and Immunology, Hanover, USA (GRID:grid.254880.3) (ISNI:0000 0001 2179 2404)
6 University of New Mexico, Pulmonary and Critical Care Medicine, Albuquerque, USA (GRID:grid.266832.b) (ISNI:0000 0001 2188 8502)