哮喘电子每日控制评分（e-DASTHMA）的开发与验证：一项真实世界直接患者数据研究

Development and validation of an electronic daily control score for asthma (e-DASTHMA): a real-world direct patient data study.

作者信息

Sousa-Pinto Bernardo, Jácome Cristina, Pereira Ana Margarida, Regateiro Frederico S, Almeida Rute, Czarlewski Wienczyslawa, Kulus Marek, Shamji Mohamed H, Boulet Louis-Philippe, Bonini Matteo, Brussino Luisa, Canonica G Walter, Cruz Alvaro A, Gemicioglu Bilun, Haahtela Tari, Kupczyk Maciej, Kvedariene Violeta, Larenas-Linnemann Desirée, Louis Renaud, Niedoszytko Marek, Pham-Thi Nhân, Puggioni Francesca, Romantowski Jan, Sastre Joaquin, Scichilone Nicola, Taborda-Barata Luis, Ventura Maria Teresa, Vieira Rafael José, Agache Ioana, Bedbrook Anna, Bergmann Karl C, Amaral Rita, Azevedo Luís Filipe, Bosnic-Anticevich Sinthia, Brusselle Guy, Buhl Roland, Cecchi Lorenzo, Charpin Denis, Loureiro Claudia Chaves, de Blay Frédéric, Del Giacco Stefano, Devillier Philippe, Jassem Ewa, Joos Guy, Jutel Marek, Klimek Ludger, Kuna Piotr, Laune Daniel, Luna Pech Jorge, Makela Mika, Morais-Almeida Mario, Nadif Rachel, Neffen Hugo E, Ohta Ken, Papadopoulos Nikolaos G, Papi Alberto, Pétré Benoit, Pfaar Oliver, Yeverino Daniela Rivero, Cordeiro Carlos Robalo, Roche Nicolas, Sá-Sousa Ana, Samolinski Boleslaw, Sheikh Aziz, Ulrik Charlotte Suppli, Usmani Omar S, Valiulis Arunas, Vandenplas Olivier, Vieira-Marques Pedro, Yorgancioglu Arzu, Zuberbier Torsten, Anto Josep M, Fonseca João A, Bousquet Jean

机构信息

MEDicina da Comunidade, Informação e Decisão em Saúde, Department of Community Medicine, Information and Health Decision Sciences, Faculty of Medicine, University of Porto, Porto, Portugal; Centro de Investigação em Tecnologias e Serviços de Saúde, Rede de Investigação em Saúde, Health Research Network, MEDCIDS, Faculty of Medicine, University of Porto, Porto, Portugal; Patient Centred Innovation and Technology, Centro de Investigação em Tecnologias e Serviços de Saúde, Centre for Health Technology and Services Research, University of Porto, Porto, Portugal.

出版信息

Lancet Digit Health. 2023 Apr;5(4):e227-e238. doi: 10.1016/S2589-7500(23)00020-1. Epub 2023 Mar 3.

DOI:10.1016/S2589-7500(23)00020-1

PMID:36872189

Abstract

BACKGROUND

Validated questionnaires are used to assess asthma control over the past 1-4 weeks from reporting. However, they do not adequately capture asthma control in patients with fluctuating symptoms. Using the Mobile Airways Sentinel Network for airway diseases (MASK-air) app, we developed and validated an electronic daily asthma control score (e-DASTHMA).

METHODS

We used MASK-air data (freely available to users in 27 countries) to develop and assess different daily control scores for asthma. Data-driven control scores were developed based on asthma symptoms reported by a visual analogue scale (VAS) and self-reported asthma medication use. We included the daily monitoring data from all MASK-air users aged 16-90 years (or older than 13 years to 90 years in countries with a lower age of digital consent) who had used the app in at least 3 different calendar months and had reported at least 1 day of asthma medication use. For each score, we assessed construct validity, test-retest reliability, responsiveness, and accuracy. We used VASs on dyspnoea and work disturbance, EQ-5D-VAS, Control of Allergic Rhinitis and Asthma Test (CARAT), CARAT asthma, and Work Productivity and Activity Impairment: Allergy Specific (WPAI:AS) questionnaires as comparators. We performed an internal validation using MASK-air data from Jan 1 to Oct 12, 2022, and an external validation using a cohort of patients with physician-diagnosed asthma (the INSPIRERS cohort) who had had their diagnosis and control (Global Initiative for Asthma [GINA] classification) of asthma ascertained by a physician.

FINDINGS

We studied 135 635 days of MASK-air data from 1662 users from May 21, 2015, to Dec 31, 2021. The scores were strongly correlated with VAS dyspnoea (Spearman correlation coefficient range 0·68-0·82) and moderately correlated with work comparators and quality-of-life-related comparators (for WPAI:AS work, we observed Spearman correlation coefficients of 0·59-0·68). They also displayed high test-retest reliability (intraclass correlation coefficients range 0·79-0·95) and moderate-to-high responsiveness (correlation coefficient range 0·69-0·79; effect size measures range 0·57-0·99 in the comparison with VAS dyspnoea). The best-performing score displayed a strong correlation with the effect of asthma on work and school activities in the INSPIRERS cohort (Spearman correlation coefficients 0·70; 95% CI 0·61-0·78) and good accuracy for the identification of patients with uncontrolled or partly controlled asthma according to GINA (area under the receiver operating curve 0·73; 95% CI 0·68-0·78).

INTERPRETATION

e-DASTHMA is a good tool for the daily assessment of asthma control. This tool can be used as an endpoint in clinical trials as well as in clinical practice to assess fluctuations in asthma control and guide treatment optimisation.

FUNDING

None.

摘要

背景

经过验证的问卷用于根据报告评估过去1 - 4周的哮喘控制情况。然而，它们无法充分捕捉症状波动患者的哮喘控制情况。我们利用用于气道疾病的移动气道哨兵网络（MASK - air）应用程序，开发并验证了一种电子每日哮喘控制评分（e - DASTHMA）。

方法

我们使用MASK - air数据（27个国家的用户可免费获取）来开发和评估不同的哮喘每日控制评分。基于视觉模拟量表（VAS）报告的哮喘症状和自我报告的哮喘药物使用情况，开发了数据驱动的控制评分。我们纳入了所有年龄在16 - 90岁（在数字同意年龄较低的国家为13岁以上至90岁）的MASK - air用户的每日监测数据，这些用户在至少3个不同的日历月使用了该应用程序，并且报告了至少1天的哮喘药物使用情况。对于每个评分，我们评估了结构效度、重测信度、反应性和准确性。我们使用关于呼吸困难和工作干扰的VAS、EQ - 5D - VAS、过敏性鼻炎和哮喘控制测试（CARAT）、CARAT哮喘以及工作生产力和活动损害：过敏特异性（WPAI:AS）问卷作为对照。我们使用2022年1月1日至10月12日期间的MASK - air数据进行内部验证，并使用一组经医生诊断为哮喘的患者（INSPIRERS队列）进行外部验证，这些患者的哮喘诊断和控制情况（全球哮喘防治创议[GINA]分类）已由医生确定。

结果

我们研究了2015年5月21日至2021年12月31日期间1662名用户的135635天MASK - air数据。这些评分与VAS呼吸困难评分高度相关（斯皮尔曼相关系数范围为0.68 - 0.82），与工作对照和生活质量相关对照中度相关（对于WPAI:AS工作，我们观察到斯皮尔曼相关系数为0.59 - 0.68）。它们还显示出高重测信度（组内相关系数范围为0.79 - 0.95）和中等到高反应性（相关系数范围为0.69 - 0.79；与VAS呼吸困难相比，效应量测量范围为0.57 - 0.99）表现最佳的评分与INSPIRERS队列中哮喘对工作和学校活动的影响高度相关（斯皮尔曼相关系数为0.70；95%置信区间为0.61 - 0.78），并且在根据GINA识别未控制或部分控制哮喘患者方面具有良好的准确性（受试者工作特征曲线下面积为0.73；95%置信区间为0.68 - 0.78）。