Pate Alexander, Barrowman Michael, Webb David, Pimenta Jeanne M, Davis Kourtney J, Williams Rachael, Van Staa Tjeerd, Sperrin Matthew
Farr Institute, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK.
Real World Evidence and Epidemiology, GlaxoSmithKline, Uxbridge, UK.
BMJ Open Respir Res. 2018 Oct 25;5(1):e000339. doi: 10.1136/bmjresp-2018-000339. eCollection 2018.
Traditional phase IIIb randomised trials may not reflect routine clinical practice. The Salford Lung Study in chronic obstructive pulmonary disease (SLS COPD) allowed broad inclusion criteria and followed patients in routine practice. We assessed whether SLS COPD approximated the England COPD population and evidence for a Hawthorne effect.
This observational cohort study compared patients with COPD in the usual care arm of SLS COPD (2012-2014) with matched non-trial patients with COPD in England from the Clinical Practice Research Datalink database. Generalisability was explored with baseline demographics, clinical and treatment variables; outcomes included COPD exacerbations in adjusted models and pretrial versus peritrial comparisons.
Trial participants were younger (mean, 66.7 vs 71.1 years), more deprived (most deprived quintile, 51.5% vs 21.4%), more current smokers (47.5% vs 32.1%), with more severe Global initiative for chronic Obstructive Lung Disease stages but less comorbidity than non-trial patients. There were no material differences in other characteristics. Acute COPD exacerbation rates were high in the trial population (98.37th percentile).
The trial population was similar to the non-trial COPD population. We observed some evidence of a Hawthorne effect, with more exacerbations recorded in trial patients; however, the largest effect was observed through behavioural changes in patients and general practitioner coding practices.
传统的IIIb期随机试验可能无法反映常规临床实践。索尔福德慢性阻塞性肺疾病研究（SLS COPD）采用了宽泛的纳入标准，并在常规实践中对患者进行随访。我们评估了SLS COPD是否能代表英格兰慢性阻塞性肺疾病患者群体以及是否存在霍桑效应的证据。
这项观察性队列研究将SLS COPD常规治疗组（2012 - 2014年）中的慢性阻塞性肺疾病患者与来自临床实践研究数据链数据库的英格兰匹配的非试验慢性阻塞性肺疾病患者进行了比较。通过基线人口统计学、临床和治疗变量探讨了可推广性；结局包括调整模型中的慢性阻塞性肺疾病急性加重以及试验前与试验期间的比较。
试验参与者更年轻（平均年龄，66.7岁对71.1岁），贫困程度更高（最贫困五分位数，51.5%对21.4%），当前吸烟者更多（47.5%对32.1%），慢性阻塞性肺疾病全球倡议分级更严重，但合并症比非试验患者少。其他特征没有实质性差异。试验人群中慢性阻塞性肺疾病急性加重率较高（第98.37百分位数）。
试验人群与非试验慢性阻塞性肺疾病人群相似。我们观察到了一些霍桑效应的证据，试验患者中记录到的急性加重更多；然而，最大的效应是通过患者的行为改变和全科医生的编码实践观察到的。
