Abstract
BACKGROUND: Dupilumab has demonstrated therapeutic benefits and improved quality of life (QoL) in children with uncontrolled asthma. This real-world study-the first in a Latin-American pediatric population-evaluated its impact on asthma control and QoL in children with moderate-to-severe uncontrolled type 2 asthma and their caregivers. METHODS: We conducted a retrospective observational study of patients aged 4-16 years with moderate-to-severe uncontrolled type 2 asthma (blood eosinophils ≥150 cells/μL or fractional exhaled nitric oxide ≥20 ppb). Eleven patients received dupilumab and 12 received conventional inhaled therapy, with 52-week follow-up. Asthma control was assessed using the 7-item Asthma Control Questionnaire interviewer-administered version (ACQ-7-IA). QoL was evaluated using the Pediatric Asthma Quality of Life Questionnaire interviewer-administered version (PAQLQ[S]-IA) and the Pediatric Asthma Caregiver's Quality of Life Questionnaire (PACQLQ). The primary endpoints were changes in ACQ-7-IA, PAQLQ(S)-IA, and PACQLQ scores, and the proportion achieving clinically significant improvement (≥0.5 points). RESULTS: Dupilumab significantly improved ACQ-7-IA scores from week 4, sustained through week 52 (least squares mean difference -0.44, 95% CI -0.59 to -0.30; p < 0.001). At week 52, 86% of dupilumab-treated patients achieved clinically significant improvement versus 75% with conventional therapy (p = 0.041). Well-controlled asthma (ACQ-7-IA ≤0.75) was achieved in 73% versus 36% (p < 0.003). Dupilumab also improved PAQLQ(S)-IA and PACQLQ scores from week 24, with 100% of patients and caregivers achieving clinically significant improvement at week 52. CONCLUSIONS: In Latin-American children with moderate-to-severe type 2 asthma, dupilumab was associated with early and sustained improvements in asthma control and QoL for both patients and caregivers, supporting its role as an effective therapeutic option in real-world pediatric practice.