Optimizing treatmen in severe pediatric asthma using clinical characteristics and non-invasive (bio)markers

This thesis focuses on improving the treatment of severe asthma in children and explores the use of patient-reported outcome measures (PROMs) and non-invasive (bio)markers to better tailor treatments and support clinical decision-making. Key goals include understanding the current therapeutic landscape, and assessing PROMs and non-invasive biomarkers for asthma phenotyping.
Biologics—such as omalizumab, mepolizumab, and dupilumab—target immune pathways but are often used without clear markers to guide selection or measure efficacy, especially in children. In the PANDA study, a real-life study population of children aged 6-17 with severe asthma, we identified multiple drivers and inhibitors for pediatric pulmonologists in starting biologics. Additionally, we showed a clinical need for the initiation of biologics in children suffering from steroid toxicity, which is currently not addressed in the GINA treatment recommendation. In the PANDA cohort we also identified that fatigue was highly prevalent amongst children with severe asthma, and showed its association with asthma-related quality of life, demonstrating that fatigue is an important of patient-reported outcome. The thesis also introduces SPIRITAS, a cost-effective breath sampling device for analyzing volatile organic compounds (VOCs), which can help identify asthma phenotypes. VOC analysis proved effective in distinguishing controlled from uncontrolled asthma in multiple cohorts of children with moderate to severe asthma.
Overall, this research promotes personalized asthma care, advocating for standardized, multifactorial assessments and enhanced collaboration between researchers, clinicians, and patients to translate findings into practice.