Di Cicco Maria Elisa, Peroni Diego, Marseglia Gian Luigi, Licari Amelia
Section of Pediatrics, Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy.
Pediatric Unit, Department of Clinical, Surgical, Diagnostic and Pediatric Science, University of Pavia, Pavia, Italy.
Paediatr Drugs. 2025 Mar 22. doi: 10.1007/s40272-025-00694-6.
Pediatric asthma remains a prevalent and challenging chronic condition globally, affecting quality of life and imposing significant burdens on families and healthcare systems. Despite advancements in understanding asthma pathophysiology and treatment, key controversies persist in optimizing management strategies. Inhaled corticosteroids (ICS) are the cornerstone of treatment, reducing inflammation and preventing exacerbations. While concerns about growth suppression exist, evidence suggests that this effect is primarily associated with high doses and prolonged use, rather than standard maintenance therapy. Nonetheless, adherence to ICS remains suboptimal, necessitating strategies to ensure effective and sustained treatment. The introduction of maintenance and reliever therapy (MART) with ICS-formoterol has offered improved outcomes by simplifying regimens and reducing reliance on short-acting beta-agonists (SABA). However, evidence supporting MART and ICS-SABA regimens in younger children is limited, highlighting gaps in pediatric-focused research. Biologics targeting inflammatory pathways, such as omalizumab, mepolizumab, and dupilumab, represent a personalized approach for severe asthma but face challenges including high costs, limited long-term safety data, and uncertainty regarding their ability to modify disease progression. In addition, the complexity of treatment decisions is compounded by insufficient biomarkers and age-specific evidence to guide therapy. Addressing these gaps requires robust clinical studies and improved adherence strategies tailored to pediatric populations. This review critically examines current pharmacological strategies, unresolved issues, and evolving approaches in asthma management, emphasizing the need for personalized and evidence-based care. Enhancing treatment outcomes for pediatric asthma necessitates balancing therapeutic benefits with minimal adverse effects and leveraging ongoing research to inform future practice.
小儿哮喘在全球范围内仍是一种普遍且具有挑战性的慢性疾病，影响生活质量，并给家庭和医疗系统带来巨大负担。尽管在理解哮喘病理生理学和治疗方面取得了进展，但在优化管理策略方面仍存在关键争议。吸入性糖皮质激素（ICS）是治疗的基石，可减轻炎症并预防病情加重。虽然存在对生长抑制的担忧，但有证据表明，这种影响主要与高剂量和长期使用有关，而非标准维持治疗。尽管如此，ICS的依从性仍然不理想，需要采取策略以确保有效和持续的治疗。引入ICS-福莫特罗维持和缓解治疗（MART）通过简化治疗方案和减少对短效β受体激动剂（SABA）的依赖，带来了更好的治疗效果。然而，支持MART和ICS-SABA方案用于年幼儿童的证据有限，凸显了以儿童为重点的研究存在差距。针对炎症途径的生物制剂，如奥马珠单抗、美泊利单抗和度普利尤单抗，代表了一种针对重度哮喘的个性化方法，但面临包括高成本、长期安全性数据有限以及其改变疾病进展能力的不确定性等挑战。此外，治疗决策的复杂性因缺乏足够的生物标志物和针对特定年龄的证据来指导治疗而加剧。解决这些差距需要开展强有力的临床研究，并制定针对儿童群体的改进依从性策略。本综述批判性地审视了哮喘管理中的当前药理学策略、未解决的问题和不断发展的方法，强调了个性化和循证护理的必要性。提高小儿哮喘的治疗效果需要在治疗益处与最小不良反应之间取得平衡，并利用正在进行的研究为未来实践提供信息。
