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开发哮喘医疗负担评分作为SARP III和U-BIOPRED中严重程度的衡量指标及缓解的预测指标:来自两个主要哮喘纵向队列的结果

Development of an asthma health-care burden score as a measure of severity and predictor of remission in SARP III and U-BIOPRED: results from two major longitudinal asthma cohorts.

作者信息

Zein Joe G, Zounemat-Kerman Nazanin, Adcock Ian M, Hu Bo, Attaway Amy, Castro Mario, Dahlén Sven-Erik, Denlinger Loren C, Erzurum Serpil C, Fahy John V, Gaston Benjamin, Hastie Annette T, Israel Elliot, Jarjour Nizar N, Levy Bruce D, Mauger David T, Moore Wendy, Peters Michael C, Sumino Kaharu, Townsend Elizabeth, Woodruff Prescott, Ortega Victor E, Wenzel Sally E, Meyers Deborah A, Chung Kian Fan, Bleecker Eugene R

机构信息

Division of Pulmonary Medicine, Department of Medicine, Mayo Clinic, Scottsdale, AZ, USA.

Data Science Institute and National Heart & Lung Institute, Imperial College London, London, UK.

出版信息

Lancet Respir Med. 2025 Jan;13(1):35-46. doi: 10.1016/S2213-2600(24)00250-9. Epub 2024 Nov 22.

Abstract

BACKGROUND

Current asthma guidelines, including those of the European Respiratory Society (ERS) and American Thoracic Society (ATS), suboptimally predict asthma remission, disease severity, and health-care utilisation. We aimed to establish a novel approach to assess asthma severity based on asthma health-care burden data.

METHODS

We analysed prospectively collected data from the Severe Asthma Research Program III (SARP III; USA) and the European Unbiased Biomarkers for the Prediction of Respiratory Disease Outcomes (U-BIOPRED; 11 European countries) to calculate a composite burden score based on asthma exacerbations and health-care utilisation, which was modified to include the use of short-acting beta agonists (SABAs) to reflect asthma symptom burden.

FINDINGS

In SARP III, 528 adult participants with asthma were followed up for a mean of 4·4 (SD 1·6) years, and 312 (59%) had severe asthma according to the ERS-ATS definition. Among the 205 participants with asthma who used rescue SABAs daily, 90 used these two or more times a day. In U-BIOPRED, 509 adult participants with asthma were followed up for 1 year, and 421 (83%) had severe asthma. The burden score was less than 1·29 per patient-year in 106 (34%) of 312 SARP III participants and in 80 (19%) of 421 U-BIOPRED participants with severe asthma. By contrast, the burden score was above the median value in 58 (28%) SARP III and 24 (27%) U-BIOPRED participants with non-severe asthma. In both cohorts, the burden score negatively correlated with lung function, asthma control, and quality of life. A burden score of 0·15 or lower predicted asthma remission with a sensitivity greater than 91% and a specificity of 99%.

INTERPRETATION

Our findings highlight considerable discrepancies between the current definition of asthma severity and our burden score. Although the definition of severe asthma proposed by the ERS-ATS and the and Global Initiative for Asthma (GINA) is based on prescribed asthma medications, our personalised health-care burden score includes patient-centred data that reflect disease severity and accurately predicts asthma remission. Subject to prospective validation, the burden score could help to optimise the management of high-risk individuals with asthma.

FUNDING

SARP III: US National Heart, Lung, and Blood Institute; AstraZeneca; Boehringer Ingelheim; Genentech; GlaxoSmithKline; Sanofi Genzyme/Regeneron; and Teva Pharmaceuticals.

U-BIOPRED: Innovative Medicines Initiative Joint Undertaking (EU's Seventh Framework Programme and European Federation of Pharmaceutical Industries and Associations) and eTRIKS project.

摘要

背景

当前的哮喘指南,包括欧洲呼吸学会(ERS)和美国胸科学会(ATS)的指南,在预测哮喘缓解、疾病严重程度和医疗保健利用方面存在不足。我们旨在基于哮喘医疗保健负担数据建立一种评估哮喘严重程度的新方法。

方法

我们分析了前瞻性收集的来自重度哮喘研究项目III(SARP III;美国)和欧洲呼吸疾病结局预测无偏生物标志物研究(U-BIOPRED;11个欧洲国家)的数据,以根据哮喘急性加重和医疗保健利用情况计算综合负担评分,并对其进行修改以纳入短效β受体激动剂(SABA)的使用情况,以反映哮喘症状负担。

结果

在SARP III中,528名成年哮喘患者平均随访了4.4(标准差1.6)年,根据ERS-ATS定义,其中312名(59%)患有重度哮喘。在205名每天使用急救SABA的哮喘患者中,90名每天使用两次或更多次。在U-BIOPRED中,509名成年哮喘患者随访了1年,421名(83%)患有重度哮喘。在312名SARP III重度哮喘患者中,106名(34%)的负担评分低于每年1.29,在421名U-BIOPRED重度哮喘患者中,80名(19%)的负担评分低于每年1.29。相比之下,在58名(28%)SARP III非重度哮喘患者和24名(27%)U-BIOPRED非重度哮喘患者中,负担评分高于中位数。在两个队列中,负担评分与肺功能、哮喘控制和生活质量呈负相关。负担评分为0.15或更低时预测哮喘缓解的敏感性大于91%,特异性为99%。

解读

我们的研究结果突出了当前哮喘严重程度定义与我们的负担评分之间的显著差异。尽管ERS-ATS和全球哮喘防治创议(GINA)提出的重度哮喘定义基于规定的哮喘药物,但我们的个性化医疗保健负担评分包括以患者为中心的数据,这些数据反映了疾病严重程度并能准确预测哮喘缓解。经过前瞻性验证后,负担评分有助于优化哮喘高危个体的管理。

资助

SARP III:美国国立心肺血液研究所;阿斯利康;勃林格殷格翰;基因泰克;葛兰素史克;赛诺菲基因公司/再生元公司;以及梯瓦制药公司。

U-BIOPRED:创新药物倡议联合事业(欧盟第七框架计划和欧洲制药工业协会联合会)和eTRIKS项目。

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