Abstract Introduction Asthma is the most common chronic childhood disease and a leading cause of emergency visits and hospitalizations. Despite advances in therapy, a proportion of children continue to experience poor asthma control. Dupilumab, a monoclonal antibody targeting the interleukin-4 receptor alpha (IL-4Rα) and inhibiting both IL-4 and IL-13 signaling, represents a novel approach for type 2 (T2) asthma. Dupilumab has demonstrated reductions in exacerbations and improvements in lung function in children with moderate-to-severe asthma. However, real-world data on clinical response and health-care utilization outcomes in pediatric populations remain limited. We hypothesize that Dupilumab therapy will result in superior asthma control compared with traditional asthma treatments in children with asthma. Methods We conducted a retrospective cohort study of thirty-seven pediatric patients with moderate-to- severe asthma treated with Dupilumab ≥6 months. Matched-pairs analysis using paired t-tests (Asthma Control Test (ACT) scores, forced expiratory volume in one second (FEV1), forced expiratory volume in one second/forced vital capacity (FEV1/FVC)) and Wilcoxon signed-rank tests (systemic corticosteroids) assessed immediate pre-post treatment effects. Linear mixed-effects models evaluated within-patient changes across treatment phases (baseline, six to eleven months, twelve to twenty-three months, twenty-four to thirty-five months), adjusting for age and sex with patient-specific random intercepts estimated via restricted maximum likelihood. Alpha was set at 0.05. Results Among 37 pediatric patients (mean age 12.4 years, 74% Hispanic/Latino) with severe asthma and poor baseline control (ACT 15.5 ± 4.8), Dupilumab therapy resulted in progressive clinical improvements. Systemic corticosteroid use declined significantly across all phases, decreasing by approximately 3 courses per year (p = 0.03) Figure 1A. Mixed-effects models revealed ACT score increases of 3.81 points (95% CI 1.84 to 5.78) at 6-11 months, 5.02 points (2.69 to 7.36) at 12-23 months, and 8.17 points (5.55 to 10.79) at 24-35 months (all p 0.001) Figure 1B. FEV₁ improvements were modest and not statistically significant. However, FEV₁/FVC ratio improved significantly, increasing by + 6.48% (95% CI 2.12-10.85, p = 0.005) at 12-23 months and +9.54% (95% CI 4.88-14.20, p 0.001) at 24-35 months. Conclusions Dupilumab therapy led to significant and sustained improvements in asthma control and reduced systemic corticosteroid use up to 35 months of follow-up. While FEV₁ gains were modest and not statistically significant, a progressive improvement in FEV₁/FVC ratio suggests reduced airway obstruction with long-term therapy. These findings support Dupilumab as an effective long-term treatment option for children with severe asthma, particularly in improving symptom control and airflow obstruction while reducing systemic steroid burden. This abstract is funded by: None
Dhmour et al. (Fri,) studied this question.