Protocol for an open labelled observational study in children & adolescents with severe asthma with an eosinophilic

Atul Gupta1,2, Ines de Mir Messa3, Jose Valverde-Molina4

  • 1Paediatric Respiratory Department, King's College Hospital, Department of Women and Children's Health, London, United Kingdom.

Plos One
|December 19, 2025
PubMed

Insights

This study assesses the real-world effectiveness of mepolizumab in children with severe eosinophilic asthma. It aims to provide crucial long-term safety and efficacy data for this patient population.

Area of Science:

  • Pediatric Pulmonology
  • Immunology
  • Pharmacology

Background:

  • Severe pediatric eosinophilic asthma presents significant risks, including exacerbations and reduced quality of life.
  • Biologic therapies targeting eosinophilic pathways offer improved management for severe asthma.
  • Mepolizumab, an anti-interleukin-5 monoclonal antibody, is approved for severe eosinophilic asthma in children aged ≥6, but pediatric real-world data are limited.

Purpose of the Study:

  • To evaluate the long-term safety and efficacy of mepolizumab in children and adolescents with severe eosinophilic asthma in real-world clinical practice.
  • To assess mepolizumab's performance in patients with comorbidities and on concomitant medications.

Main Methods:

  • An open-label, multinational, multicenter observational cohort study (CASAM) enrolling 150 pediatric patients (6-17 years) with severe asthma.
  • Data collection over 36 months (12 months pre- and 24 months post-mepolizumab initiation) using pseudonymized electronic case report forms.
  • Primary outcome: comparison of clinically significant asthma exacerbation rates pre- and post-mepolizumab treatment.

Main Results:

  • The study is designed to compare the rates of clinically significant asthma exacerbations in the pre-exposure and 12-month post-exposure periods with mepolizumab treatment.
  • Long-term safety and efficacy data in children and adolescents, including those with comorbidities, will be analyzed.

Conclusions:

  • This study will provide valuable real-world evidence on the long-term safety and efficacy of mepolizumab in pediatric severe eosinophilic asthma.
  • Findings will inform clinical decision-making and management strategies for this vulnerable patient group.
Abstract

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