Mathioudakis Alexander G, Ananth Sachin, Bradbury Thomas, Csoma Balazs, Sivapalan Pradeesh, Stovold Elizabeth, Fernandez-Romero Gustavo, Lazar Zsofia, Criner Gerard J, Jenkins Christine, Papi Alberto, Jensen Jens-Ulrik, Vestbo Jørgen
Division of Infection, Immunity and Respiratory Medicine, School of Biological Sciences, The University of Manchester, Manchester M23 9LT, UK.
North West Lung Centre, Wythenshawe Hospital, Manchester University NHS Foundation Trust, Manchester Academic Health Science Centre, Manchester M23 9LT, UK.
Biomedicines. 2021 Dec 5;9(12):1837. doi: 10.3390/biomedicines9121837.
A recently published ERS core outcome set recommends that all trials of COPD exacerbation management should assess the treatment success (or "cure" of the exacerbation), defined as a dichotomous measure of the overall outcome of an exacerbation. This methodological systematic review describes and compares the instruments that were used to assess treatment success or failure in 54 such RCTs, published between 2006-2020. Twenty-three RCTs used composite measures consisting of several undesirable outcomes of an exacerbation, together defining an overall unfavourable outcome, to define treatment failure. Thirty-four RCTs used descriptive instruments that used qualitative or semi-quantitative descriptions to define cure, marked improvement, improvement of the exacerbation, or treatment failure. Treatment success and failure rates among patients receiving guidelines-directed treatments at different settings and timepoints are described and could be used to inform power calculations in future trials. Descriptive instruments appeared more sensitive to treatment effects compared to composite instruments. Further methodological studies are needed to optimise the evaluation of treatment success/failure. In the meantime, based on the findings of this systematic review, the ERS core outcome set recommends that cure should be defined as sufficient improvement of the signs and symptoms of the exacerbation such that no additional systemic treatments are required.
欧洲呼吸学会（ERS）最近发布的核心结局集建议，所有慢性阻塞性肺疾病（COPD）加重期管理试验均应评估治疗成功率（或加重期的“治愈”情况），其定义为加重期总体结局的二分法测量指标。本方法学系统评价描述并比较了2006年至2020年间发表的54项此类随机对照试验（RCT）中用于评估治疗成功或失败的工具。23项RCT使用了综合指标，这些指标由加重期的几个不良结局组成，共同定义总体不良结局，以此来定义治疗失败。34项RCT使用了描述性工具，这些工具使用定性或半定量描述来定义治愈、显著改善、加重期改善或治疗失败。文中描述了在不同环境和时间点接受指南指导治疗的患者的治疗成功率和失败率，这些数据可用于为未来试验的样本量计算提供参考。与综合工具相比，描述性工具似乎对治疗效果更敏感。需要进一步开展方法学研究以优化治疗成功/失败的评估。同时，基于本系统评价的结果，ERS核心结局集建议，治愈应定义为加重期的体征和症状有足够改善，以至于无需额外的全身治疗。
