Slob Elise M, Maitland-Van der Zee Anke-Hilse, Koppelman Gerard H, Pijnenburg Mariëlle W
Department of Respiratory Medicine, Amsterdam University Medical Centers, University of Amsterdam.
Pediatric Respiratory Medicine, Emma Children's Hospital, Amsterdam UMC, Amsterdam.
Curr Opin Allergy Clin Immunol. 2019 Apr;19(2):141-147. doi: 10.1097/ACI.0000000000000517.
Childhood asthma is a heterogeneous disease and many children have uncontrolled disease. Therefore an individualized approach is needed to improve asthma outcomes in children. Precision medicine using clinical characteristics, biomarkers, and the rapidly involving field of genomics and pharmacogenomics aims to achieve asthma control and reduce future risks with less side-effects in individual children with asthma.
It is not yet possible to select treatment options on clinical characteristics. Novel monoclonal antibodies are efficacious in patients with severe, eosinophilic asthma. Reduced lung function growth and early decline is a prevalent finding in children with persistent asthma. Pharmacogenetic studies have identified children at risk for cortisol suppression when using inhaled corticosteroids.
Clinical characteristics and simple biomarkers like eosinophils, IgE, and the fraction of exhaled nitric oxide may be used in clinical practice for a basic precision medicine approach, deciding which children will have the best chance to respond to inhaled corticosteroids and to the biologicals omalizumab and mepolizumab.Further application of pharmacogenomics and breathomics needs additional studies before they can be applied as tools for precision medicine in individual children with asthma.
儿童哮喘是一种异质性疾病,许多儿童的病情未得到控制。因此,需要采取个体化方法来改善儿童哮喘的治疗效果。利用临床特征、生物标志物以及快速发展的基因组学和药物基因组学领域的精准医学,旨在实现哮喘控制,并在个体哮喘患儿中减少副作用,降低未来风险。
目前尚无法根据临床特征选择治疗方案。新型单克隆抗体对重度嗜酸性粒细胞性哮喘患者有效。肺功能增长减缓及早期下降是持续性哮喘患儿中的常见表现。药物遗传学研究已确定使用吸入性糖皮质激素时存在皮质醇抑制风险的儿童。
临床特征以及嗜酸性粒细胞、免疫球蛋白E和呼出一氧化氮分数等简单生物标志物可在临床实践中用于基本的精准医学方法,以确定哪些儿童最有可能对吸入性糖皮质激素以及生物制剂奥马珠单抗和美泊利单抗产生反应。在可将药物基因组学和呼吸组学进一步应用于个体哮喘患儿的精准医学工具之前,还需要进行更多研究。