Biologic Therapy for Severe Pediatric Asthma

Summary

Severe paediatric asthma affects a minority of children but accounts for a disproportionate share of morbidity, healthcare utilisation and diminished quality of life. This condition is typified by persistent symptoms, frequent exacerbations and suboptimal control despite high-dose inhaled corticosteroids plus second controllers. Advances in the understanding of asthma endotypes have revealed distinct type 2 inflammatory pathways driven by cytokines such as interleukin-5 (IL-5), interleukin-4 (IL-4) and thymic stromal lymphopoietin (TSLP), as well as immunoglobulin E (IgE) mechanisms. Biologic therapies—monoclonal antibodies targeting these pathways—have transformed the landscape of treatment, offering precision medicine approaches. Approved agents include anti-IgE (omalizumab), anti-IL-5 (mepolizumab, benralizumab) and anti-IL-4 receptor α (dupilumab), with emerging anti-TSLP therapies under evaluation. Clinical trials and real-world studies demonstrate that biologics reduce exacerbation rates, lower eosinophil counts, improve lung function and enhance patient-reported outcomes. Selection of the optimal biologic is guided by biomarkers such as blood eosinophil levels, exhaled nitric oxide and serum IgE, alongside clinical features. Despite these advances, challenges persist in long-term safety assessment, durability of response and equitable access across healthcare settings. Ongoing research seeks to refine predictive biomarkers, extend indications to younger age groups and evaluate combination strategies, underscoring the global significance of biologic therapy in reshaping severe paediatric asthma management.

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Biologic Therapy for Severe Pediatric Asthma publication trend

The graph below shows the total number of articles in biologic therapy for severe pediatric asthma across all publications each year (not limited to Nature Index journals).

Technical terms

Endotype: A disease subtype defined by distinct molecular or immunological mechanisms.

Monoclonal antibody: A laboratory-engineered protein designed to bind a specific target antigen.

Eosinophil: A type of white blood cell involved in allergic inflammation and asthma exacerbations.

Interleukin-5 (IL-5): A cytokine that promotes the growth and activation of eosinophils.

Immunoglobulin E (IgE): An antibody isotype central to allergic reactions and mast cell activation.

Biomarker: A measurable indicator of a biological state or response to therapy.

References

  1. Burden and unmet need for specialist care in poorly controlled and severe childhood asthma in a Danish nationwide cohort. Respiratory Research (2023).
  2. Meta-analysis of the adoption of omalizumab in the treatment of pediatric allergic diseases. Heliyon (2024).
  3. Biologic Therapies in Pediatric Asthma. Journal of Personalized Medicine (2022).
  4. Long-Term Safety of Omalizumab in Children with Asthma and/or Chronic Spontaneous Urticaria: A 4-Year Prospective Study in Real Life. Journal of Personalized Medicine (2023).
  5. Severe Pediatric Asthma Therapy: Mepolizumab. Frontiers in Pediatrics (2022).

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