# Biologic therapies for severe pediatric asthma: efficacy, safety, and biomarker-guided selection

## TL;DR

Biologic therapies for severe pediatric asthma improve disease control and reduce exacerbations, especially in children with specific biomarker profiles.

## Contribution

This paper synthesizes current evidence on biologic therapies and provides clinical guidance for biomarker-driven selection in pediatric asthma.

## Key findings

- Biologics like omalizumab, mepolizumab, and dupilumab reduce exacerbations and enable steroid-sparing in selected children.
- Tezepelumab shows efficacy across biomarker strata, while benefits are greatest in T2-high profiles with elevated eosinophils or FeNO.
- Lung-function improvements are modest but clinically meaningful, though gaps remain in treatment duration and long-term safety.

## Abstract

Severe pediatric asthma is a heterogeneous, high-burden disease marked by variable corticosteroid responsiveness, frequent exacerbations, and substantial impairment in quality of life. Advances in airway immunobiology, particularly the delineation of type-2 (T2) pathways (IgE, IL-5, IL-4/IL-13) and epithelial alarmins, have enabled the development of targeted biologic therapies for biomarker-defined patient subgroups.

To synthesize current evidence on the efficacy and safety of biologic therapies for severe pediatric asthma and to translate biomarker-driven selection into practical clinical guidance, while outlining emerging therapeutic directions.

Targeted biologics, anti-IgE (omalizumab), anti-IL-5/IL-5Rα (mepolizumab, benralizumab; pediatric data for reslizumab remain limited), anti-IL-4Rα (dupilumab), and anti-TSLP (tezepelumab) improve disease control, reduce severe exacerbations, and enable steroid-sparing in appropriately selected children. Benefits are greatest in T2-high profiles, particularly with elevated blood eosinophils and/or fractional exhaled nitric oxide (FeNO), while tezepelumab shows efficacy across biomarker strata. Lung-function gains are modest to moderate but clinically meaningful. Persisting gaps include optimal treatment duration, stopping rules, long-term safety, cost, and equitable access.

Biologic therapies have reshaped the care of severe pediatric asthma, operationalizing precision medicine through immunologic endotyping and biomarker-guided selection. Priorities now include standardized definitions of response and remission, robust long-term safety data, and strategies to ensure equitable access across diverse pediatric populations.