OBJECTIVES: Dupilumab, a fully human monoclonal antibody, blocks the shared receptor component for interleukin (IL)-4 and IL-13, key and central drivers of type 2 (T2) inflammation, and demonstrated an acceptable safety profile and clinical efficacy in children aged 6–11 with uncontrolled moderate-to-severe asthma in VOYAGE (NCT02948959). This analysis assessed dupilumab efficacy by baseline (BL) inhaled corticosteroid (ICS) dose in children with T2 asthma (blood eosinophils ≥150 cells/μL or FeNO [fractional exhaled nitric oxide] ≥20 ppb [parts per billion] at VOYAGE baseline).

METHODS: Children received weight-based dupilumab 100/200 mg every 2 weeks or placebo and were stratified by medium (n = 195) or high (n = 152) BL ICS dose per GINA 2015 guidelines. In a prespecified, multiplicity-controlled analysis, annualized severe asthma exacerbation rates (AER) over the 52-week treatment period in the high ICS group were evaluated. Other prespecified analyses were AER in the medium ICS group and, in both groups, changes from BL in pre-bronchodilator (BD) percentage predicted (pp) FEV₁ and interviewer-administered 7-Item Asthma Control Questionnaire (ACQ-7-IA) score.

RESULTS: Dupilumab vs placebo significantly reduced AER by 63% (P = 0.0004) and 59% (P = 0.0028) in the high and medium ICS groups, respectively. At Week 52, dupilumab improved pre-BD ppFEV₁ in both high (LS mean difference 5.70 [0.99, 10.40]; P = 0.0180) and medium (LS mean difference 9.35 [4.38, 14.33]; P = 0.0003) ICS groups. At Week 24, dupilumab improved ACQ-7 in both high (LS mean difference -0.37 [-0.62, -0.11]; P = 0.0047) and medium (LS mean difference -0.33 [-0.56, -0.10]; P = 0.0045) ICS groups.

CONCLUSIONS: Dupilumab demonstrated clinical efficacy in children with uncontrolled moderate-to-severe T2 asthma regardless of ICS dose at baseline, with significant reductions in exacerbations as well as significant improvements in ppFEV₁ and asthma control compared to placebo.