Real-world effectiveness and safety of dupilumab therapy in children ≤6 years with uncontrolled persistent asthma: a propensity-matched retrospective cohort study

Background Asthma is common in children. Dupilumab is a biological agent that showed effectiveness in older children with uncontrolled asthma; however, its efficacy and safety are lacking among the very young children (≤6) who were excluded from clinical trials. Methods Using TriNetX, children ≤6 years old with persistent asthma who received a concomitant prescription for dupilumab for any indication, in addition to standard asthma treatment, were compared with those on standard treatment alone. Using 1:1 propensity score matching across 35 covariates, outcomes such as acute exacerbations, status asthmaticus, need for oral corticosteroid (OCS), emergency room (ER) visits, anaphylaxis, infections, and inpatient admissions were assessed at 6 and 12 months. Results Of 44,952 patients analyzed, 301 patients were matched per group. Asthmatic children with concomitant dupilumab prescriptions were associated with a reduction in acute exacerbations at 6 and 12 months (RR 0.53 and 0.46, respectively; p < 0.05), OCS use (RR 0.67 and 0.66; p < 0.05), ER visits, and infections (all p < 0.05). However, the risk of anaphylaxis was higher in cohort one at 6 and 12 months (RR 1.80 and 1.74; p < 0.05). Other outcomes, such as status asthmaticus, eosinophilia, and inpatient admissions, were similar between the cohorts. Conclusions This real-world analysis shows dupilumab therapy alongside standard treatment in children ≤6 years with persistent asthma is significantly associated with reduced acute exacerbations, OCS use, and ER visits. These findings support dupilumab's clinical benefit for this vulnerable group, aiding clinical decisions.