Abdel-Aziz Mahmoud I, Neerincx Anne H, Vijverberg Susanne J H, Hashimoto Simone, Brinkman Paul, Gorenjak Mario, Toncheva Antoaneta A, Harner Susanne, Brandstetter Susanne, Wolff Christine, Perez-Garcia Javier, Hedman Anna M, Almqvist Catarina, Corcuera-Elosegui Paula, Korta-Murua Javier, Sardón-Prado Olaia, Pino-Yanes Maria, Potočnik Uroš, Kabesch Michael, Kraneveld Aletta D, Maitland-van der Zee Anke H
Department of Respiratory Medicine, Amsterdam UMC, University of Amsterdam, 1105 AZ Amsterdam, The Netherlands.
Department of Clinical Pharmacy, Faculty of Pharmacy, Assiut University, 71526 Assiut, Egypt.
J Pers Med. 2021 May 28;11(6):484. doi: 10.3390/jpm11060484.
There is a clinical need to identify children with poor asthma control as early as possible, to optimize treatment and/or to find therapeutic alternatives. Here, we present the "Systems Pharmacology Approach to Uncontrolled Pediatric Asthma" (SysPharmPediA) study, which aims to establish a pediatric cohort of moderate-to-severe uncontrolled and controlled patients with asthma, to investigate pathophysiological mechanisms underlying uncontrolled moderate-to-severe asthma in children on maintenance treatment, using a multi-omics systems medicine approach. In this multicenter observational case-control study, moderate-to-severe asthmatic children (age; 6-17 years) were included from four European countries (Netherlands, Germany, Spain, and Slovenia). Subjects were classified based on asthma control and number of exacerbations. Demographics, current and past patient/family history, and clinical characteristics were collected. In addition, systems-wide omics layers, including epi(genomics), transcriptomics, microbiome, proteomics, and metabolomics were evaluated from multiple samples. In all, 145 children were included in this cohort, 91 with uncontrolled (median age = 12 years, 43% females) and 54 with controlled asthma (median age = 11.7 years, 37% females). The two groups did not show statistically significant differences in age, sex, and body mass index z-score distribution. Comprehensive information and diverse noninvasive biosampling procedures for various omics analyses will provide the opportunity to delineate underlying pathophysiological mechanisms of moderate-to-severe uncontrolled pediatric asthma. This eventually might reveal novel biomarkers, which could potentially be used for noninvasive personalized diagnostics and/or treatment.
尽早识别哮喘控制不佳的儿童，以优化治疗和/或寻找替代治疗方法，这具有临床必要性。在此，我们介绍“儿童难治性哮喘的系统药理学方法”（SysPharmPediA）研究，该研究旨在建立一个由中度至重度难治性和已控制的哮喘患儿组成的队列，采用多组学系统医学方法，研究维持治疗的儿童中度至重度难治性哮喘的病理生理机制。在这项多中心观察性病例对照研究中，纳入了来自四个欧洲国家（荷兰、德国、西班牙和斯洛文尼亚）的中度至重度哮喘儿童（年龄6 - 17岁）。根据哮喘控制情况和发作次数对受试者进行分类。收集人口统计学信息、当前和既往患者/家族病史以及临床特征。此外，还从多个样本中评估了全系统的组学层面，包括表观（基因组学）、转录组学、微生物组、蛋白质组学和代谢组学。该队列共纳入145名儿童，其中91名哮喘控制不佳（中位年龄 = 12岁，43%为女性），54名哮喘已得到控制（中位年龄 = 11.7岁，37%为女性）。两组在年龄、性别和体重指数z评分分布上无统计学显著差异。全面的信息和用于各种组学分析的多样非侵入性生物采样程序，将为描绘儿童中度至重度难治性哮喘的潜在病理生理机制提供机会。这最终可能揭示新的生物标志物，这些生物标志物可能用于非侵入性个性化诊断和/或治疗。
