Identified research priorities
| Research priority | Action steps |
| Identifying young patients at highest risk for progression to severe disease | Establish a European real-world paediatric severe asthma cohort with longitudinal follow-up (into adulthood), building on existing initiatives and infrastructures (e.g. SPACE, UBIOPRED, GAN, PERMEABLE, COBRAPed, VIRASTHMA2 and the Danish National Database for Severe Asthma) |
| Identifying paediatric responders and nonresponders to biologicals | Standardise a definition of a responder/nonresponder Large-scale collaboration to include paediatric patients with severe asthma/allergy Collaborative study efforts using joint research/clinical protocols (e.g. pragmatic real-world studies and comparative studies with biologics) Assess whether incorporation of identified predictive biomarkers in clinical decision-making models improve clinical outcomes and are cost-effective |
| Understanding phenotypes of severe disease, and differences between adult and paediatric severe disease phenotypes | In-depth phenotyping of severe asthma/allergy patients combining clinical characteristics and -omics data (e.g. UBIOPRED, COBRAPed, SysPharmPediA, SPACE, PERMEABLE and VIRASTHMA2) in combination with validation in adult cohorts (e.g. SHARP and UBIOPRED) Long-term follow-up of severe paediatric asthma into adulthood |
| Long-term efficacy and safety of biologics use | Establish a European real-world paediatric cohort with patients on biologics of choice and/or switchers with longitudinal follow-up (assess the maintenance dosage, long-term follow-up on overall health and QoL of patients) |
| Understanding molecular mechanisms to accelerate drug development using ex vivo translational models | Establish collaborations with immunologists and molecular biologists to ensure hypothesis/outcome validation in preclinical disease models Implement the latest molecular biology techniques (i.e. CRISPR-Cas9 and single-cell sequencing assays) both to validate existing outcomes based on associations and to discover novel cell (sub)types that mediate underlying inflammatory processes Develop noninvasive techniques to explore pathophysiological mechanisms |
| Understanding patient and caregiver perspectives on biologics use | Perform qualitative studies on patient and caregiver experiences Establish a European patient and caregiver advisory board specific for biologics use (in collaboration with European Lung Foundation and national patient organisations) Develop and apply a platform for children and parents’ involvement |
| Harmonising treatment protocols across Europe | Systematically assess which differences exist within the European countries Develop online educational programmes and regularly update these programmes based on novel scientific evidence |
| Applying a precision medicine approach in severe paediatric asthma | Evaluate the added value of biomarker and -omics data for individual treatment selection Combine disease history data, clinical measures, and biomarker data into useful disease score models |
SPACE: Severe Paediatric Asthma Collaborative in Europe; UBIOPRED: Unbiased Biomarkers in Prediction of Respiratory Disease Outcomes; GAN: Global Asthma Network; PERMEABLE: Personalized Medicine Approach for Asthma and Allergy Biologicals Selection; VIRASTHMA2: Inflammatory and Immune Profiles During a Severe Exacerbation in Preschool Asthmatic Children; COBRAPed: Pediatric Cohort of Bronchial Obstruction and Asthma; SysPharmPedia: Systems Pharmacology Approach to Difficult-to-Treat Pediatric Asthma; SHARP: Severe Heterogeneous Asthma Registry, Patient-Oriented; QoL: quality of life.