Adult Cystic Fibrosis Center, Division of Respiratory, Critical Care and Occupational Pulmonary Medicine, Department of Internal Medicine, University of Utah, 26 North Mario Capecchi Drive, Salt Lake City, UT, 84132, USA.
Intermountain Pediatric Cystic Fibrosis Center, Division of Pediatric Pulmonology, Department of Pediatrics, University of Utah, 81 North Mario Capecchi Drive, Salt Lake City, UT, 84113, USA.
BMC Med Res Methodol. 2019 Apr 26;19(1):88. doi: 10.1186/s12874-019-0705-0.
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.
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.
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.
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.
预测囊性纤维化 (CF) 疾病结局的炎症生物标志物将提高临床试验的效力，并有助于更好地对临床评估进行个性化处理。具有代表性的患者队列将有助于寻找可信、可推广、可重复和准确的生物标志物。
我们从 Mountain West CF 联合会 (MWCFC) 护理中心招募患者，进行前瞻性观察性研究，以评估痰液炎症生物标志物。在获得知情同意后，各中心随机招募了具有 CF 且临床稳定的痰液生产者作为研究对象，年龄在 12 岁及以上，且无先前的器官移植史。
从 2014 年 12 月 8 日至 2016 年 1 月 16 日，我们共招募了 114 名 CF 患者（53 名男性），这些患者持续提供数据。基线特征包括平均年龄 27 岁（标准差 [SD]=12）、预计第 1 秒用力呼气量占预计值的 80%（SD=23%）、前一年有 1.0 次肺部加重（SD=1.2）、海拔高度为 328 米（SD=112）。与 2014 年美国 CF 基金会患者登记处（CFFPR）中的其他患者相比，MWCFC 患者的性别、年龄、肺功能、体重和加重发作率、糖尿病、胰腺功能不全、CF 相关关节炎以及包括耐甲氧西林金黄色葡萄球菌、铜绿假单胞菌、洋葱伯克霍尔德菌复合体、真菌和非结核分枝杆菌感染在内的气道感染的分布情况相似。他们使用 CF 特异性治疗的频率也相似。
MWCFC 中的随机选择、产生痰液的患者代表了 CFFPR 中的产生痰液的患者。他们具有相似的特征、肺功能和肺部加重发作率、微生物感染和 CF 特异性治疗的使用情况。这些发现将合理地使未来对炎症生物标志物的定量测量的解释推广到 CFFPR 中的产生痰液的患者。
