Omalizumab for pediatric allergic asthma: evidence-based insights on efficacy and safety from a meta-analysis of randomized trials

OBJECTIVES: 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. METHODS: 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 I2 statistics. RESULTS: 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; I2 = 51.6%), SAEs (RR = 0.56, 95% CI: 0.35-0.90; I2 = 43.1%), and hospitalizations (RR = 0.35, 95% CI: 0.22-0.56; I2 = 0.0%). No significant difference was found in TEAEs (RR = 0.96, 95% CI: 0.92-1.01; I2 = 1.5%). Evidence regarding lung function and asthma control was limited and could not be quantitatively pooled. CONCLUSION: 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. SYSTEMATIC REVIEW REGISTRATION: https://www.crd.york.ac.uk/PROSPERO/view/CRD420251132811, identifer CRD420251132811.