Systems Pharmacology Approach to Uncontrolled Pediatric Asthma
- Conditions
- Pediatric AsthmaUncontrolled Asthma
- Registration Number
- NCT04865575
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 145
General:
- 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.
- 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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Saliva and stool microbiome Baseline Differences in 16S rRNA microbial α- and ß- diversities between cases and controls
Exhaled breath metabolome Baseline Differences in presence and levels of volatile organic compounds between cases and controls
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (6)
University Regensburg
🇩🇪Regensburg, Germany
University of Maribor
🇸🇮Maribor, Slovenia
Universidad de La Laguna
🇪🇸Santa Cruz De Tenerife, Spain
University of the Basque Country
🇪🇸San Sebastián, Spain
Karolinska University Hospital
🇸🇪Stockholm, Sweden
Utrecht University
🇳🇱Utrecht, Netherlands