BACKGROUND We have previously developed Core Outcome Measures sets for Severe Asthma (COMSA) by multi-stakeholder consensus. There are no patient-centred tools to quantify response to biologics for severe asthma. We aimed to develop paediatric and adult CompOsite iNdexes For Response in asthMa (CONFiRM) incorporating clinical parameters and patient-reported quality of life (QoL). METHODS International expert healthcare professionals (HCPs) and patients with severe asthma were invited to: 1) develop consensus levels of clinically relevant changes for each outcome measure within COMSA; 2) use multicriteria decision analysis to develop the CONFiRM scores and 3) assess their internal validity. A separate group of HCPs evaluated CONFiRM's external validity. RESULTS Five levels of change for each COMSA outcome were agreed. Severe exacerbations and maintenance oral corticosteroids use were rated as most important in determining both paediatric and adult CONFiRM scores. There was strong agreement between HCPs and patients, although patients assigned greater importance to QoL. The CONFiRM score quantified response to a biological from -31 (deterioration) to 69 (best possible response). Paediatric and adult CONFiRMs had good discriminative ability for a sufficient (AUC≥0.92) and a substantial (AUC≥0.95) response to biologics. Both CONFiRMs demonstrated excellent external validity (Spearman correlation coefficients 0.9 and 0.8 for paediatric and adult respectively (p<0.0001)). CONCLUSIONS We have developed novel patient-centred paediatric and adult CONFiRMs which include QoL measures. CONFiRMs should allow a more holistic understanding of response for the patient and a standardised assessment of the effectiveness of biologics between studies. Further research is needed to prospectively validate CONFiRM scores.

中文翻译：

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以患者为中心的综合评分作为评估儿童和成人严重哮喘对生物治疗反应的工具。


背景 我们之前已经根据多方利益相关者共识开发了严重哮喘核心结果测量集 （COMSA）。没有以患者为中心的工具来量化严重哮喘对生物制剂的反应。我们旨在开发儿科和成人 CompOsite iNdexes for Response in asthMa （CONFiRM），结合临床参数和患者报告的生活质量 （QoL）。方法 邀请国际专家医疗保健专业人员 （HCP） 和严重哮喘患者： 1） 为 COMSA 中的每个结果测量制定临床相关变化的共识水平;2） 使用多标准决策分析来制定 CONFiRM 分数，以及 3） 评估其内部效度。另一组 HCP 评估了 CONFiRM 的外部效度。结果 商定了每个 COMSA 结局的 5 个变化水平。在确定儿科和成人 CONFiRM 评分时，严重加重和维持口服皮质类固醇的使用被评为最重要。HCP 和患者之间有很强的一致性，尽管患者更重视 QoL。CONFiRM 评分量化了对生物的反应，从 -31（恶化）到 69（最佳反应）。儿童和成人 CONFiRMs 对生物制剂的充分 （AUC≥0.92） 和实质性 （AUC≥0.95） 反应具有良好的区分能力。两种 CONFiRM 均表现出优异的外部效度 （儿童和成人的 Spearman 相关系数分别为 0.9 和 0.8 （p<0.0001））。结论 我们开发了以患者为中心的新型儿科和成人 CONFiRM，其中包括 QoL 测量。CONFiRM 应允许更全面地了解患者的反应，并在研究之间对生物制剂的有效性进行标准化评估。 需要进一步的研究来前瞻性验证 CONFiRM 评分。