A multistakeholder Delphi consensus core outcome set for clinical trials in moderate-to-severe asthma (coreASTHMA)
A multistakeholder Delphi consensus core outcome set for clinical trials in moderate-to-severe asthma (coreASTHMA)
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DOI：
10.1016/j.anai.2021.03.022
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发表时间：
2021-06-18
影响因子：
5.9
通讯作者：
Moloney, Rachael M.
中科院分区：
文献类型：
--
作者：
Tejwani, Vickram;Chang, Hsing-Yuan;Moloney, Rachael M.
背景：长期控制哮喘的治疗方法已经得到改善，包括一类有前途但昂贵的生物疗法。然而，评估这些和其他新疗法的临床试验使用了各种不同的结果来评估疗效。哮喘护理的发展要求对对患者和其他利益相关者最重要的结果进行重新检查。目的：为中重度哮喘患者的3期和4期临床药物试验提供一组核心结果。方法：我们使用了一个强大而深入的多利益相关者共识过程，将患者、临床医生、监管机构、支付方、卫生技术评估人员、研究人员和产品开发人员聚集在一起，就结果达成共识。我们使用了一种改进的德尔菲法来达成共识，该方法改编自有效性试验倡议中的核心结果测量方法，与核心结果集开发的当代方法标准保持一致。结果：以下结果被纳入最终核心组：严重哮喘加重、哮喘控制改变、哮喘特异性或严重哮喘特异性生活质量、哮喘特异性住院时间（即在任何级别护理中住院24小时以上）或入院，以及哮喘特异性急诊科就诊。结论：这5个结果代表了用于中重度哮喘的3期和4期临床药物试验的最小核心结果集。无论是否包括其他异质性的主要或次要结局，将这些结局作为最低限度的一致收集，将允许在临床试验中对哮喘治疗的效果进行有意义的比较。(C) 2021美国过敏、哮喘与免疫学学会。Elsevier Inc.出版。版权所有。
Background: Treatments for long-term control of asthma have improved and include a promising but expensive class of biologic therapies. However, the clinical trials evaluating these and other novel treatments have used a variety of different outcomes to evaluate efficacy. The evolution of asthma care calls for a re-examination of outcomes that are most important to patients and other stakeholders.Objective: To develop a core set of outcomes to be measured in phase 3 and phase 4 clinical drug trials in patients with moderate-to-severe asthma.Methods: We used a robust and in-depth multistakeholder consensus process bringing together patients, clinicians, regulators, payers, health technology assessors, researchers, and product developers to reach consensus on outcomes. We used a modified Delphi method to reach consensus, an approach adapted from the Core Outcome Measures in Effectiveness Trials Initiative aligned with contemporary methodological standards for core outcome set development.Results: The following outcomes were included in the final core set: severe asthma exacerbation, change in asthma control, asthma-specific or severe asthma-specific quality of life, asthma-specific hospital stay (ie, >24hour stays at any level of care) or admission, and asthma-specific emergency department visit.Conclusion: These 5 outcomes represent a minimum set of core outcomes for use in phase 3 and phase 4 clinical drug trials in moderate-to-severe asthma. Consistent collection of these outcomes as minimum, independent of whether additional heterogeneous primary or secondary outcomes are included, will allow for meaningful comparisons of the effect of asthma therapies across clinical trials. (C) 2021 American College of Allergy, Asthma & Immunology. Published by Elsevier Inc. All rights reserved.