Omalizumab for pediatric allergic asthma: evidence-based insights on efficacy and safety from a meta-analysis of randomized trials
Abstract
This meta-analysis aimed to synthesize data from English-language randomized controlled trials (RCTs) to evaluate the efficacy and safety of omalizumab in patients aged 6–20 years with allergic asthma, with specific attention to age-stratified outcomes where feasible. Conducted in accordance with the PRISMA 2020 guidelines, this review followed a pre-registered protocol (PROSPERO: CRD420251132811). Eligible studies were English-language RCTs involving predominantly pediatric and adolescent patients with allergic asthma; one included trial enrolled participants up to 20 years of age, but only 15 participants (0.8% of the total sample) were aged 18–20 years, and all remaining participants were <18 years. Studies compared omalizumab with control treatment (placebo or standard therapy). Efficacy outcomes included asthma exacerbations, forced expiratory volume in 1 s (FEV1), and Childhood Asthma Control Test (C-ACT) scores. Safety outcomes encompassed treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and asthma-related hospitalizations. Data were pooled using random-effects models where appropriate, with heterogeneity assessed via I 2 statistics. Six RCTs involving 1,835 participants (omalizumab: 1,134; control: 701) aged 6–20 years were included. Omalizumab significantly reduced asthma exacerbations (relative risk RR = 0.66, 95% CI: 0.55-0.79; I 2 = 51.6%), SAEs (RR = 0.56, 95% CI: 0.35–0.90; I 2 = 43.1%), and hospitalizations (RR = 0.35, 95% CI: 0.22–0.56; I 2 = 0.0%). No significant difference was found in TEAEs (RR = 0.96, 95% CI: 0.92–1.01; I 2 = 1.5%). Evidence regarding lung function and asthma control was limited and could not be quantitatively pooled. Omalizumab is effective in reducing exacerbations, SAEs, and hospitalizations in pediatric and adolescent allergic asthma, with a favorable safety profile. Evidence regarding lung function and asthma control remains limited and should be interpreted cautiously. These findings support its use as an add-on therapy for moderate-to-severe disease inadequately controlled by standard treatments. https://www.crd.york.ac.uk/PROSPERO/view/CRD420251132811 , identifer CRD420251132811.