Section of Pulmonary Medicine, 5C, Dartmouth-Hitchcock Medical Center, One Medical Center Drive, Lebanon, NH, 03756, USA.
Division of Pulmonary, Critical Care and Sleep Medicine, University of Kansas Medical Center, 3901 Rainbow Boulevard, Mailstop 3007, Kansas City, KS, 66160, USA.
Sci Rep. 2021 Mar 1;11(1):4897. doi: 10.1038/s41598-021-84041-y.
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.
囊性纤维化（CF）患者群体试图解释肺部感染加重（PEX）治疗效果的异质性。血清和痰液中的炎症介质可能可以识别 CF 患者（PwCF）中对治疗反应不佳的风险人群。然而，由于缺乏对这些介质与反应表型之间的明确关联，限制了其在临床上的应用。在这项初步研究中，我们通过评估 PEX 期间的健康相关生活质量（HRQoL），前瞻性地对治疗反应表型进行了特征描述。我们还测量了血清和痰液中的肺功能和与铁相关的生化参数。我们根据初始改善后症状评分是否恶化，将患者分为持续症状缓解者（SRs）和非持续症状缓解者（NSRs）。我们使用线性混合模型（LMM）来确定肺功能、血液学、血清和痰液炎症指标的趋势在两组患者之间是否存在差异。在 20 名 PwCF 患者中，我们根据肺功能、PEX 发作时和治疗持续时间无显著差异，确定了 10 名 SRs 和 10 名 NSRs。在 PEX 期间，SRs 的肺功能预测趋势好于 NSRs。SRs 和 NSRs 之间的血清和痰液铁水平的非线性趋势存在显著差异。在 CF 成年患者中，PEX 治疗反应表型可能与血清和痰液铁浓度的独特趋势相关。
