Protocol for an open labelled observational study in children & adolescents with severe asthma with an eosinophilic phenotype treated with mepolizumab (CASAM)

Introduction Children with severe eosinophilic asthma are at an increased risk of severe exacerbations, medication side effects, impaired lung function, and significantly reduced quality of life. The advent of biologic agents aimed at eosinophilic pathways has significantly improved management optio...

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Detalhes bibliográficos
Autores: Gupta, A., De-Mir-Messa, I., Valverde-Molina, J., Murray, C.S., Moral, L., Borrego, J.T., Pike, K., Díaz-Izquierdo, A., Martínez-Cañavate, A., Nagakumar, P., Cook, J., Gupta, L., Aliaga, Y., Callejón, A.C., Aguilera, P.C., Corullón, S.C., Elosegui, P.C., de la Serna-Blázquez, O., Garcia, C.D., Izquierdo, A.D., Gáboli, M., Gimeno-Díaz de Atauri, A.G.D., López-Neyra, A., Blasco, J.L., Cañavate, A.M., Requena, L.M., Morón, J.N., Navarrete, L.V., Turnbull, A., Nwokoro, C., Tang, A.
Tipo de documento: artigo
Estado:Versão publicada
Data de publicação:2025
País:España
Recursos:Institut d'Investigació i Innovació Parc Taulí (I3PT)
Repositório:r-I3PT. Repositorio Institucional Producción Científica del Institut d'Investigació i Innovació Parc Taulí
OAI Identifier:oai:i3pt.fundanetsuite.com:p6935
Acesso em linha:https://i3pt.portalinvestigacion.com/publicaciones/6935
https://www.scopus.com/inward/record.uri?eid=2-s2.0-105025378094&doi=10.1371%2Fjournal.pone.0335399&partnerID=40&md5=46d704ca94cc0444c0e1162cdffd7e37
Access Level:Acceso aberto
Palavra-chave:corticosteroid
mepolizumab
antiasthmatic agent
monoclonal antibody
adolescent
allergic rhinitis
Article
atopic dermatitis
child
clinical assessment
clinical outcome
cohort analysis
demographics
disease exacerbation
disease severity
drug efficacy
drug safety
electronic medical record
eosinophilic asthma
female
food allergy
headache
health care
health care personnel
hospitalization
human
hyperventilation
information processing
lung function
major clinical study
male
multicenter study
observational study
pediatric patient
pharmacokinetics
phenotype
rhinopharyngitis
risk assessment
severe asthma
sociodemographics
socioeconomic parameters
treatment outcome
upper abdominal pain
asthma
drug effect
drug therapy
eosinophil
eosinophilia
pathology
quality of life
Adolescent
Anti-Asthmatic Agents
Antibodies, Monoclonal, Humanized
Asthma
Child
Eosinophilia
Eosinophils
Female
Humans
Male
Observational Studies as Topic
Phenotype
Quality of
Descrição
Resumo:Introduction Children with severe eosinophilic asthma are at an increased risk of severe exacerbations, medication side effects, impaired lung function, and significantly reduced quality of life. The advent of biologic agents aimed at eosinophilic pathways has significantly improved management options for patients with severe asthma. Mepolizumab, an anti-interleukin-5 monoclonal antibody, was approved in 2018 as an add-on maintenance treatment for patients with severe eosinophilic asthma aged =6 years of age. Mepolizumab’s efficacy and safety is well documented in adults, but paediatric data remain limited. This real-world observational study will evaluate the long-term safety and efficacy of mepolizumab in children and adolescents, including those with comorbidities and on concomitant medications to provide a clearer picture of its performance in everyday clinical practice. Methods and analysis Design: CASAM is an open label, multinational (Spain and United Kingdom), multicenter (15 Spanish and 6 UK public institutions) observational cohort study. Sample size: 150 paediatric patients on mepolizumab for SA between 6–17 years of age will be enrolled. Study Duration: Study will be conducted over 36 months (12 months pre mepolizumab initiation and 24 months after initiation). Data Collection: Pseudonymized data (prospective and retrospective) from medical charts will be collected and entered in the electronic case report forms (eCRFs) of the electronic data capture (EDC) system. Primary Outcome: To compare the rates of clinically significant asthma exacerbations in the pre-exposure and the 12-month post-exposure period with mepolizumab treatment. Protocol Version: CASAM Study_Protocol_V2.0_16th November 2023 KCH © 2025 Gupta et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

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