Pneumology Department, Hospital Universitario y Politécnico La Fe, Valencia, Spain.
Servicio de Neumología, Hospital Santa Creu i Sant Pau, Barcelona, Spain.
Expert Rev Respir Med. 2023 Apr;17(4):279-293. doi: 10.1080/17476348.2023.2205125. Epub 2023 Apr 20.
Bronchiectasis is a very heterogeneous disease. This heterogeneity has several consequences: severity cannot be measured by a single variable, so multidimensional scores have been developed to capture it more broadly. Some groups of patients with similar clinical characteristics or prognoses (clinical phenotypes), and even similar inflammatory profiles (endotypes), have been identified, and these have been shown to require a more specific treatment.
We comment on this 'stratified' model of medicine as an intermediate step toward the application of the usual concepts on which precision medicine is based (such as cellular, molecular or genetic biomarkers, treatable traits and individual clinical fingerprinting), whereby each subject presents certain specific characteristics and receives individualized treatment.
True precision or personalized medicine is based on concepts that have not yet been fully achieved in bronchiectasis, although some authors are already beginning to adapt them to this disease in terms of pulmonary and extrapulmonary etiologies, clinical fingerprinting (specific to each individual), cellular biomarkers such as neutrophils and eosinophils (in peripheral blood) and molecular biomarkers such as neutrophil elastase. In therapeutic terms, the future is promising, and some molecules with significant antibiotic and anti-inflammatory properties are being developed.
支气管扩张症是一种非常异质性的疾病。这种异质性有几个后果:严重程度不能用单一变量来衡量,因此开发了多维评分来更广泛地捕捉它。已经确定了一些具有相似临床特征或预后(临床表型)的患者群体,甚至具有相似炎症特征(表型)的患者群体,并且已经表明这些患者需要更具体的治疗。
我们评论了这种“分层”医学模型,作为向应用基于精准医学的常用概念(如细胞、分子或遗传生物标志物、可治疗特征和个体临床特征)的中间步骤,其中每个个体都具有某些特定特征并接受个体化治疗。
真正的精准或个性化医学基于尚未在支气管扩张症中完全实现的概念,尽管一些作者已经开始根据肺部和肺外病因、临床特征(针对每个个体)、中性粒细胞和嗜酸性粒细胞等细胞生物标志物(外周血)以及中性粒细胞弹性蛋白酶等分子生物标志物来适应这种疾病。在治疗方面,未来前景广阔,一些具有显著抗生素和抗炎特性的分子正在开发中。