Changes in physiological, functional and structural markers of cystic fibrosis lung disease with treatment of a pulmonary exacerbation
Changes in physiological, functional and structural markers of cystic fibrosis lung disease with treatment of a pulmonary exacerbation
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DOI：
10.1136/thoraxjnl-2012-202538
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发表时间：
2013-06-01
期刊：
影响因子：
10
通讯作者：
Alton, Eric W. F. W.
中科院分区：
文献类型：
--
作者：
Horsley, Alex R.;Davies, Jane C.;Alton, Eric W. F. W.
背景囊性纤维化(CF)的临床试验因缺乏明确的和临床相关的结果指标而受到阻碍。目的在多中心环境下评估一系列传统的和新型的囊性肺疾病生物标记物，作为选择CFTR基因治疗临床试验的结果分析的贡献研究。方法一项多中心观察性研究，对因医生定义的肺症状加重而接受治疗的成人和儿童CF患者进行研究。测量在开始时和静脉注射抗生素疗程后立即进行。结果1 S患者的用力呼气量明显改善(P<0.05)。
Background Clinical trials in cystic fibrosis (CF) have been hindered by the paucity of well characterised and clinically relevant outcome measures.Aim To evaluate a range of conventional and novel biomarkers of CF lung disease in a multicentre setting as a contributing study in selecting outcome assays for a clinical trial of CFTR gene therapy.Methods A multicentre observational study of adult and paediatric patients with CF (>10 years) treated for a physician-defined exacerbation of CF pulmonary symptoms. Measurements were performed at commencement and immediately after a course of intravenous antibiotics. Disease activity was assessed using 46 assays across five key domains: symptoms, lung physiology, structural changes on CT, pulmonary and systemic inflammatory markers.Results Statistically significant improvements were seen in forced expiratory volume in 1 s (p