Prospective multicenter randomized patient recruitment and sample collection to enable future measurements of sputum biomarkers of inflammation in an observational study of cystic fibrosis

Theodore G. Liou, Frederick R. Adler, Natalia Argel, Fadi Asfour, Perry S. Brown, Barbara A. Chatfield, Cori L. Daines, Dixie Durham, Jessica A. Francis, Barbara Glover, Theresa Heynekamp, John R. Hoidal, Judy L. Jensen, Ruth Keogh, Carol M. Kopecky, Noah Lechtzin, Yanping Li, Jerimiah Lysinger, Osmara Molina, Craig NakamuraKristyn A. Packer, Katie R. Poch, Alexandra L. Quittner, Peggy Radford, Abby J. Redway, Scott D. Sagel, Shawna Sprandel, Jennifer L. Taylor-Cousar, Jane B. Vroom, Ryan Yoshikawa, John P. Clancy, J. Stuart Elborn, Kenneth N. Olivier, David R. Cox

Research output: Contribution to journalArticle

2 Scopus citations

Abstract

Background: Biomarkers of inflammation predictive of cystic fibrosis (CF) disease outcomes would increase the power of clinical trials and contribute to better personalization of clinical assessments. A representative patient cohort would improve searching for believable, generalizable, reproducible and accurate biomarkers. Methods: We recruited patients from Mountain West CF Consortium (MWCFC) care centers for prospective observational study of sputum biomarkers of inflammation. After informed consent, centers enrolled randomly selected patients with CF who were clinically stable sputum producers, 12 years of age and older, without previous organ transplantation. Results: From December 8, 2014 through January 16, 2016, we enrolled 114 patients (53 male) with CF with continuing data collection. Baseline characteristics included mean age 27 years (SD = 12), 80% predicted forced expiratory volume in 1 s (SD = 23%), 1.0 prior year pulmonary exacerbations (SD = 1.2), home elevation 328 m (SD = 112) above sea level. Compared with other patients in the US CF Foundation Patient Registry (CFFPR) in 2014, MWCFC patients had similar distribution of sex, age, lung function, weight and rates of exacerbations, diabetes, pancreatic insufficiency, CF-related arthropathy and airway infections including methicillin-sensitive or -resistant Staphylococcus aureus, Pseudomonas aeruginosa, Burkholderia cepacia complex, fungal and non-tuberculous Mycobacteria infections. They received CF-specific treatments at similar frequencies. Conclusions: Randomly-selected, sputum-producing patients within the MWCFC represent sputum-producing patients in the CFFPR. They have similar characteristics, lung function and frequencies of pulmonary exacerbations, microbial infections and use of CF-specific treatments. These findings will plausibly make future interpretations of quantitative measurements of inflammatory biomarkers generalizable to sputum-producing patients in the CFFPR.

Original languageEnglish (US)
Article number88
JournalBMC medical research methodology
Volume19
Issue number1
DOIs
StatePublished - Apr 26 2019

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Keywords

  • Calprotectin
  • Cystic Fibrosis Foundation patient registry
  • Cystic fibrosis
  • HMGB-1
  • Neutrophil elastase
  • Randomized observational trial
  • Sputum inflammation
  • Study design

ASJC Scopus subject areas

  • Epidemiology
  • Health Informatics

Cite this

Liou, T. G., Adler, F. R., Argel, N., Asfour, F., Brown, P. S., Chatfield, B. A., Daines, C. L., Durham, D., Francis, J. A., Glover, B., Heynekamp, T., Hoidal, J. R., Jensen, J. L., Keogh, R., Kopecky, C. M., Lechtzin, N., Li, Y., Lysinger, J., Molina, O., ... Cox, D. R. (2019). Prospective multicenter randomized patient recruitment and sample collection to enable future measurements of sputum biomarkers of inflammation in an observational study of cystic fibrosis. BMC medical research methodology, 19(1), [88]. https://doi.org/10.1186/s12874-019-0705-0