Department of Allergology-Centre de l'Asthme et des Allergies, Hôpital d'Enfants Armand Trousseau, Assistance Publique-Hôpitaux de Paris, Paris, France.
UMR-S 1136, Equipe EPAR, INSERM Sorbonne Université, Institut Pierre Louis d'Epidémiologie et de Santé Publique, Paris, France.
Pediatr Allergy Immunol. 2019 Mar;30(2):159-171. doi: 10.1111/pai.13007. Epub 2018 Dec 16.
Asthma is no longer considered as a single disease but rather as a syndrome corresponding to different entities and pathophysiologic pathways. A targeted strategy is part of personalized medicine which aims to better define each patient's phenotype and endotype so as to prescribe the most suitable treatment at an individual level. Omalizumab and, more recently, mepolizumab are the first biologics approved for children (6-18 years). Omalizumab is now widely used to treat severe allergic asthma in children and is highly effective for asthma exacerbations and asthma control with a good safety profile. Moreover, several other drugs-lebrikizumab, dupilumab, tezepelumab, mepolizumab, reslizumab, benralizumab-are used or are being studied in both teenagers and adults and could benefit younger children in the near future. We hypothesize that defining the asthma phenotype/endotype regarding the type and intensity of inflammation, association with allergic or non-allergic comorbidities, and airway remodeling should contribute to the choice of a specific biologic. Pediatric specificities have to be addressed and validated by studies in children. Long-term effectiveness and particularly the impact on the natural history of asthma should also be investigated. Severe asthma in children is a complex disease, and patients have to be referred to a specialized pediatric asthma center to confirm diagnosis and initiate the best treatment strategy which could include biologics while taking into account their high cost.
哮喘不再被认为是一种单一的疾病,而是一种与不同实体和病理生理途径相对应的综合征。靶向策略是个性化医学的一部分,旨在更好地定义每个患者的表型和内型,以便在个体层面上开出最合适的治疗方案。奥马珠单抗和最近的美泊利单抗是首批批准用于儿童(6-18 岁)的生物制剂。奥马珠单抗现已广泛用于治疗儿童严重过敏性哮喘,对哮喘加重和哮喘控制非常有效,安全性良好。此外,还有其他几种药物-利布雷珠单抗、度普利尤单抗、特泽普单抗、美泊利单抗、雷西珠单抗、贝那利珠单抗-在青少年和成人中使用或正在研究中,在不久的将来可能会使年幼的儿童受益。我们假设,根据炎症的类型和强度、与过敏或非过敏合并症的关联以及气道重塑来定义哮喘表型/内型,将有助于选择特定的生物制剂。儿科的特殊性需要通过儿童研究来证实。长期疗效,特别是对哮喘自然史的影响,也需要进行研究。儿童严重哮喘是一种复杂的疾病,患者必须转介到专门的儿科哮喘中心,以确认诊断并启动最佳治疗策略,其中可能包括生物制剂,同时考虑到其高昂的成本。
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