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

John R. Hoidal¹  Judy L. Jensen¹  Theodore G. Liou¹  Kristyn A. Packer¹  Jane B. Vroom¹  Jessica A. Francis¹  Yanping Li¹  Abby J. Redway²  Theresa Heynekamp²  Peggy Radford³  Natalia Argel³  Barbara Glover⁴  Ryan Yoshikawa⁴  Craig Nakamura⁴  Ruth Keogh⁵  Carol M. Kopecky⁶  Scott D. Sagel⁶  Frederick R. Adler⁷  Cori L. Daines⁸  Osmara Molina⁸  John P. Clancy⁹  Noah Lechtzin^1,10  Jennifer L. Taylor-Cousar^1,11  Katie R. Poch^1,11  J. Stuart Elborn^1,12  Alexandra L. Quittner^1,13  Barbara A. Chatfield^1,14  Fadi Asfour^1,14  Kenneth N. Olivier^1,15  Shawna Sprandel^1,16  Jerimiah Lysinger^1,16  David R. Cox^1,17  Perry S. Brown^1,18  Dixie Durham^1,18 

[1] Adult Cystic Fibrosis Center, Division of Respiratory, Critical Care and Occupational Pulmonary Medicine, Department of Internal Medicine, University of Utah;Adult Cystic Fibrosis Program, Division of Pulmonary, Critical Care and Sleep Medicine, DoIM MSC10-5550, 1 University of New Mexico;Cystic Fibrosis Center, Phoenix Children’s Hospital;Cystic Fibrosis Center;Department of Medical Statistics, London School of Hygiene and Tropical Medicine;Department of Pediatrics, Children’s Hospital Colorado, University of Colorado School of Medicine;Departments of Mathematics, University of Utah;Division of Pediatric Pulmonary and Sleep Medicine, Department of Pediatrics, University of Arizona Health Sciences;Division of Pulmonary Medicine, Department of Pediatrics, University of Cincinnati;Division of Pulmonary and Critical Care and Sleep Medicine, Department of Medicine, Johns Hopkins University School of Medicine;Division of Pulmonary and Critical Care and Sleep Medicine, Department of Medicine, National Jewish Health;Faculty of Medicine, Health and Life Sciences, Queen’s University Belfast;Former: Department of Psychology, University of Miami;Intermountain Pediatric Cystic Fibrosis Center, Division of Pediatric Pulmonology, Department of Pediatrics, University of Utah;Laboratory of Chronic Airway Infection, Pulmonary Branch, National Heart Lung and Blood Institute, National Institutes of Health;Montana Cystic Fibrosis Center;Nuffield College;St. Luke’s Cystic Fibrosis Center of Idaho;

关键词: Cystic fibrosis;    Randomized observational trial;    Study design;    Sputum inflammation;    HMGB-1;    Neutrophil elastase;   

DOI  :  10.1186/s12874-019-0705-0

来源: DOAJ

【 摘 要 】

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.

【 授权许可】

Unknown