Systems Pharmacology Approach to Uncontrolled Pediatric Asthma (SysPharmPediA)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 145
- 试验地点
- 6
- 主要终点
- Saliva and stool microbiome
研究概览
简要总结
Background:
Asthma is a heterogeneous respiratory disease and the most common chronic disease in children. A small subset of children has continuous poor asthma control despite appropriate adherence to asthma medication. There is a clinical need to identify these children as early as possible to optimize treatment and/or to find therapeutic alternatives. Therefore, the "Systems Pharmacology approach to uncontrolled Pediatric Asthma" (SysPharmPediA) study was set up.
Objective:
To establish a cohort of pediatric moderate-to-severe uncontrolled and controlled patients with asthma in order to investigate pathophysiological mechanisms underlying uncontrolled moderate-to-severe asthma in children on maintenance treatment, using a multi-omics systems medicine approach.
Methods:
In this multicenter observational case-control study, moderate-to-severe asthmatic children (n=145, age 6-17 years), were included in specialized hospitals in four European countries (Netherlands, Germany, Spain and Slovenia). Recruited subjects were selected based on good asthma control (controlled asthmatics, n=54) or poor asthma control / recurrent exacerbations (uncontrolled asthmatics, n=91). Comprehensive details concerning 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 will be evaluated from multiple collected, relatively non-invasive, samples of from the recruited individuals, such as: blood, feces, saliva, nasal swabs and exhaled breath. Follow-up visits were performed 6 and 12 months after inclusion.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Prospective
入排标准
- 年龄范围
- 6 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Between 6-17 years of age
- •Doctor diagnosis of asthma
- •In addition to the general criteria, a case with uncontrolled asthma (case group A) should meet:
- •Asthma treatment Global INitiative of Asthma (GINA) step 3 or higher
- •≥ 1of the following criteria (based on the guideline 'Childhood Asthma' of the section paediatric pulmonology of the Dutch Society of Paediatricians, 2007):
- •Frequent exacerbations requiring OCS use (≥1 in the past year) and/or
- •Severe exacerbations requiring hospitalization or ER visits in the past year and/or
- •ACQ/ACT scores indicating uncontrolled asthma
- •In addition to the general criteria, a case with an acute exacerbation (case group B) should meet:
- •Asthma treatment Global INitiative of Asthma (GINA) step 2 or higher
- •Current severe asthma exacerbation requiring hospitalization
- •In addition to the general inclusion criteria, a control (controlled asthma) should meet the following criteria:
- •Asthma treatment Global INitiative of Asthma (GINA) step 3 or higher
- •Lack of severe exacerbations requiring OCS use or hospitalizations or ER visits in the past year
- •ACQ/ACT scores indicating well controlled asthma or few reported asthma symptoms during asthma checks in the past year.
排除标准
- •Recent use of a course of antibiotics (< 1 month). This will affect the microbiome analyses.
- •A patient can be re-screened 1 month after the use of the antibiotic treatment has finished.
结局指标
主要结局
Saliva and stool microbiome
时间窗: Baseline
Differences in 16S rRNA microbial α- and ß- diversities between cases and controls
Exhaled breath metabolome
时间窗: Baseline
Differences in presence and levels of volatile organic compounds between cases and controls
次要结局
未报告次要终点
研究者
Prof. dr. A.H. Maitland-van der Zee
Professor
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)