Tables
- TABLE 1
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-effectiveUnderstanding 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 adulthoodLong-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 mechanismsUnderstanding 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’ involvementHarmonising 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 evidenceApplying 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 modelsSPACE: 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.
Supplementary Materials
Supplementary Material
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Supplementary tables 00381-2021.supplement
Vol 7 Issue 4
Table of Contents
Expert meeting report: towards a joint European roadmap to address the unmet needs and priorities of paediatric asthma patients on biologic therapy
