Efficacy of seven biologics for paediatric asthma: a systematic review and meta-analysis

Background Biologic therapies have transformed the management of severe asthma; however, paediatric evidence remains limited and uneven across agents. We evaluated the efficacy and safety of biologic therapies in children and adolescents with moderate-to-severe asthma. Methods We searched PubMed, Embase, Web-of-Science, and Cochrane Library for randomized controlled trials (RCTs) enrolling patients <18 years receiving biologic add-on therapy versus placebo. Primary outcomes were severe asthma exacerbations and adverse events (AEs). Secondary outcomes included asthma control, quality of life, and lung function. Risk of bias was assessed using RoB2. Meta-analyses were conducted when outcomes were sufficiently homogeneous, with heterogeneity quantified using I 2 . Results 18 RCTs and 1 post-hoc pooled analysis met inclusion criteria; 10 contributed to the quantitative synthesis. Year-round omalizumab reduced exacerbation risk by 41% (RR 0.59 [0.49–0.71]), whereas seasonal prophylaxis did not significantly reduce fall/winter exacerbations (OR 0.30 [0.07–1.26]); the reduction was significant only under a fixed-effect model (OR 0.39 [0.22–0.72]). Dupilumab reduced severe exacerbations by 41% (rate ratio 0.59 [0.36–0.98]) and consistently improved lung function up to week 52. Mepolizumab reduced exacerbations by 27% (rate ratio 0.73 [0.56–0.95]). Evidence for benralizumab, lebrikizumab, tezepelumab and depemokimab was limited to single trials and inconclusive. AE rates were comparable between biologic and placebo groups. Conclusion Biologic therapies reduce exacerbation burden in children and adolescents with moderate-to-severe asthma, with acceptable short-term safety; evidence is strongest for omalizumab, dupilumab, and mepolizumab. The paediatric evidence base remains limited and uneven; dedicated trials and head-to-head comparisons are needed.