Kostikas Konstantinos, Greulich Timm, Mackay Alexander J, Lossi Nadine S, Aalamian-Mattheis Maryam, Nunez Xavier, Pagano Veronica A, Patalano Francesco, Clemens Andreas, Vogelmeier Claus F
Respiratory Medicine Dept, University of Ioannina, Ioannina, Greece.
Dept of Medicine, Pulmonary and Critical Care Medicine, University Medical Center Giessen and Marburg, Philipps-Universität Marburg, Member of the German Center for Lung Research (DZL), Giessen, Germany.
ERJ Open Res. 2019 Feb 25;5(1). doi: 10.1183/23120541.00243-2018. eCollection 2019 Feb.
The association between clinically relevant changes in patient-reported outcomes (PROs) and forced expiratory volume in 1 s (FEV) in patients with chronic obstructive pulmonary disease (COPD) has rarely been investigated. Using CRYSTAL, a 12-week open-label study in symptomatic, nonfrequently exacerbating patients with moderate COPD, we assessed at baseline the correlations between several PROs (Baseline Dyspnoea Index, modified Medical Research Council dyspnoea scale, COPD Assessment Test (CAT) and Clinical COPD Questionnaire (CCQ)), and between FEV and PROs. Associations between clinically relevant responses in FEV, CAT, CCQ and Transition Dyspnoea Index (TDI) at week 12 were also assessed. Using data from 4324 patients, a strong correlation was observed between CAT and CCQ (r=0.793) at baseline, with moderate or weak correlations between other PROs, and no correlation between FEV and any PRO. At week 12, 2774 (64.2%) patients were responders regarding TDI, CAT or CCQ, with 583 (13.5%) responding using all three measures. In comparison, 3235 (74.8%) were responders regarding FEV, TDI, CAT or CCQ, with 307 (7.1%) responding concerning all four parameters. Increases in lung function were accompanied by clinically relevant improvements of PROs in a minority of patients. Our results also suggest that PROs are not interchangeable. Thus, the observed treatment success in a clinical trial may depend on the selected parameters.
慢性阻塞性肺疾病（COPD）患者报告结局（PROs）的临床相关变化与1秒用力呼气容积（FEV）之间的关联鲜有研究。我们利用CRYSTAL（一项针对中度COPD有症状、不频繁加重患者的为期12周的开放标签研究），在基线时评估了几种PROs（基线呼吸困难指数、改良医学研究委员会呼吸困难量表、慢性阻塞性肺疾病评估测试（CAT）和临床慢性阻塞性肺疾病问卷（CCQ））之间的相关性，以及FEV与PROs之间的相关性。还评估了第12周时FEV、CAT、CCQ和过渡性呼吸困难指数（TDI）的临床相关反应之间的关联。利用4324例患者的数据，在基线时观察到CAT与CCQ之间存在强相关性（r=0.793），其他PROs之间存在中度或弱相关性，FEV与任何PRO之间无相关性。在第12周时，2774例（64.2%）患者在TDI、CAT或CCQ方面有反应，其中583例（13.5%）在所有三项指标上均有反应。相比之下，3235例（74.8%）患者在FEV、TDI、CAT或CCQ方面有反应，307例（7.1%）在所有四项参数上均有反应。少数患者肺功能改善伴随着PROs的临床相关改善。我们的结果还表明，PROs不可互换。因此，临床试验中观察到的治疗成功可能取决于所选参数。
