Figures
- FIGURE 1
The number of children who were not prescribed a biologic and who had suboptimal asthma control based on the Severe Paediatric Asthma Collaborative in Europe criteria, eligible for omalizumab, mepolizumab and dupilumab.
- FIGURE 2
The number of children who were prescribed omalizumab with suboptimal asthma control based on the Severe Paediatric Asthma Collaborative in Europe criteria who were eligible to switch to a different biologic. For two children, eligibility to switch was unclear due to lack of data on eosinophil count. One child with no option to switch had <2 exacerbations (therefore ineligible for mepolizumab) and was <12 years old (therefore ineligible for dupilumab).
Tables
- TABLE 1
Summary of Severe Paediatric Asthma Collaborative in Europe participating centres and the number of children with severe asthma recruited from each centre
Hospital City, country Number of children recruited Anna Meyer Children's University Hospital Florence, Italy 10 Emma Children's Hospital AMC Amsterdam, The Netherlands 9 Erasmus MC-Sophia Children's Hospital Rotterdam, The Netherlands 11 Marien Hospital Wesel, Germany 11 Marmara University Faculty of Medicine Pendik Hospital Pendik, Turkey 2 Oslo University Hospital Oslo, Norway 7 Sapienza University of Rome Rome, Italy 5 Royal Brompton Hospital London, UK 10 Royal Hospital for Sick Children Edinburgh, UK 6 Royal London Hospital London, UK 9 - TABLE 2
Eligibility criteria for the three currently licensed biologics, based on manufacturers’ recommendations
Biologics Licensed eligibility criteria Omalizumab 1. Age ≥6 years 2. Uncontrolled asthma with frequent symptoms and multiple documented severe asthma exacerbations despite daily high-dose inhaled corticosteroids, plus a long-acting inhaled β-agonist# 3. IgE mediated asthma (positive skin-prick test and/or raised specific IgE). 4. For age ≥12 years, reduced lung function (FEV1<80%) 5. Total IgE over 30 and up to 700 or 1300 IU·mL−1 according to age Mepolizumab 1. Age ≥6 years 2. Baseline blood eosinophils ≥150 cells·μL−1 3. ≥2 exacerbations a year (systemic corticosteroids use, unplanned medical visits/hospital admissions) Dupilumab 1. Aged ≥12 years 2. Eosinophilic/type 2 asthma 3. Or, oral corticosteroid dependent or with comorbid moderate-to-severe atopic dermatitis #: We defined uncontrolled asthma as an asthma control test score of ≤19, or “partly controlled/uncontrolled” using the Global Initiative for Asthma assessment questions, or having ≥2 exacerbations a year, or a combination. FEV1: forced expiratory volume in 1 s.
- TABLE 3
Summary demographics and findings of all recruited children. Demographics, background, investigation results, asthma control scores and respiratory treatments for all participants
Registry variables Number of patients Age 6–11 years; mean±sem 26 (32%); 9.37±0.31 years ≥12 years; mean±sem 54 (68%); 14.70±0.22 years Overall mean±sem 12.96±0.33 years Sex male/female 49 (61%)/ 31 (39%) Ethnicity Caucasians: 60 (75%);
South-east Asians: 8 (10%);
Black: 8 (10%);
Mixed: 4 (5%)Parental history Asthma: 38;
Atopic eczema: 17;
Allergic rhinitis: 33Smoking history Personal smoking: 2;
Paternal smoking: 41;
Maternal smoking: 12Allergic asthma Skin prick tests Positive for perennial aeroallergen: 31;
Positive for food allergen: 14Specific IgE Positive for perennial aeroallergen: 42;
Positive for food allergen: 16Highest blood eosinophils (cells·µL-1) in the 6 months prior to enrolment ≥150: 41;
<150: 11;
Not recently assessed: 28Highest blood IgE (IU·mL−1) in the 6 months prior to enrolment ≥700: 21;
30–699: 26;
<30: 5;
Not recently assessed: 28Highest FeNO (ppb) in the 6 months prior to enrolment >35: 28;
20–35: 11;
<20: 16;
Not recently assessed: 25Asthma control ACT score Median (IQR) ACT 20 (16–23) Control by ACT Poor control (<20): 26;
Good control (≥20): 31;
Not assessed: 23GINA asthma control Uncontrolled: 25;
Partly controlled: 23;
Well controlled: 32≥2 exacerbations per year Total:49;
With good ACT/GINA control: 12Suboptimal control by SPACE definition 62 Best FEV1 (% predicted) in the 6 months prior to enrolment <70%: 7;
70–79%: 10;
80–89%: 16;
≥90%: 46;
Not recently assessed: 1Data are presented as n (%) or n, unless otherwise stated. FeNO: exhaled nitric oxide fraction; ACT: asthma control test; IQR: interquartile range; GINA: Global Initiative for Asthma; SPACE: Severe Paediatric Asthma Collaborative in Europe; FEV1: forced expiratory volume in 1 s.
- TABLE 4
Therapies in addition to high-dose inhaled corticosteroids (ICSs) and long-acting β2 agonist (LABA).
Nonbiologic addition Biologic addition Number of children Montelukast 15 Tiotropium 4 Ipratropium 1 Triamcinolone 1 Montelukast, Tiotropium 1 Montelukast, Tiotropium Ipratropium 1 Omalizumab 10 Montelukast Omalizumab 15 Ipratropium Omalizumab 1 Montelukast, Tiotropium Omalizumab 4 Montelukast, Ipratropium Omalizumab 1 Mepolizumab 5 Montelukast Mepolizumab 2 Tiotropium Mepolizumab 1 Ipratropium Mepolizumab 1 Montelukast Dupilumab 1 Tiotropium Dupilumab 1 All children were receiving a high dose of inhaled corticosteroids (ICSs), as defined by the SPACE protocol [4]. 15 children were on ICS and LABA alone.
