D3.126 - Evaluation of Severe Pediatric Asthma Response to Biologics Using the Pediatric CONFiRM Score: A Real-Life Cohort Analysis

Biologics have improved outcomes in severe pediatric asthma, yet response assessment remains difficult to standardize because benefits span multiple clinical and patient-reported domains. The Pediatric CompOsite iNdex For Response in asthMa (CONFiRM) integrates severe exacerbations, maintenance oral corticosteroid (OCS) dose, asthma control (ACT/cACT), lung function (FEV₁ % predicted), and quality of life (PAQLQ). We assessed real-world multidimensional response to biologic therapy using Pediatric CONFiRM, explored domain-specific contributions and responder dynamics over time, and compared responses among omalizumab, dupilumab, and mepolizumab.

We performed a retrospective observational study at the Pediatric Allergy and Pulmonology Unit, Fondazione IRCCS Policlinico San Matteo, University of Pavia. Children with severe asthma who initiated omalizumab, dupilumab, or mepolizumab and completed ≥12 months of follow-up were included. Pediatric CONFiRM scores were calculated at baseline (T0), 6 months (T1), and 12 months (T2). Patients were classified as substantial responders (>45 points), sufficient responders (25–45), or insufficient responders (<25).

Thirty-three children were included (median age 11 years; 51.5% male). At baseline, disease control was poor despite high-intensity therapy (ACT/cACT 18 [15–20], PAQLQ 5.2 [4.5–6.1], FEV₁ 94% predicted [79–102]), with a median of 2 [1–3] severe exacerbations in the prior 12 months. At T1, total CONFiRM scores ranged from −2 to 51 (median 34 [23–41]); 67% were sufficient responders, 6% substantial, and 27% insufficient. At T2, scores increased (median 39 [28–45], range 0–51), with substantial responders rising to 24%, sufficient to 55%, and insufficient decreasing to 21%, indicating that ~80% achieved a clinically meaningful response after one year. Exacerbation reduction was the main driver, with >50% achieving complete elimination. ACT/cACT and PAQLQ improved significantly (median +5 and +0.8; both p<0.001), whereas FEV₁ changes were modest and non-significant. Maintenance OCS contributed minimally due to rare chronic use. No significant differences emerged across biologics. From T1 to T2, 27% improved category, 63.6% remained stable, and 9.1% declined.

Pediatric CONFiRM is feasible and clinically useful for real-world standardized monitoring of biologic response in severe pediatric asthma, capturing progressive multidomain benefits and supporting individualized treatment decisions.