Sardon-Prado Olaia, Diaz-Garcia Carolina, Corcuera-Elosegui Paula, Korta-Murua Javier, Valverde-Molina Jose, Sanchez-Solis Manuel
Division of Paediatric Respiratory Medicine, Donostia University Hospital, 20014 San Sebastián, Spain.
Department of Pediatrics, University of the Basque Country (UPV/EHU), 20014 Leioa, Spain.
J Clin Med. 2023 Sep 8;12(18):5846. doi: 10.3390/jcm12185846.
Recognition of phenotypic variability in pediatric asthma allows for a more personalized therapeutic approach. Knowledge of the underlying pathophysiological and molecular mechanisms (endotypes) of corresponding biomarkers and new treatments enables this strategy to progress. Biologic therapies for children with severe asthma are becoming more relevant in this sense. The T2 phenotype is the most prevalent in childhood and adolescence, and non-T2 phenotypes are usually rare. This document aims to review the mechanism of action, efficacy, and potential predictive and monitoring biomarkers of biological drugs, focusing on the pediatric population. The drugs currently available are omalizumab, mepolizumab, benralizumab, dupilumab, and 1ezepelumab, with some differences in administrative approval prescription criteria between the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Previously, we described the characteristics of severe asthma in children and its diagnostic and therapeutic management.
认识到儿童哮喘的表型变异性有助于采取更个性化的治疗方法。了解相应生物标志物和新治疗方法的潜在病理生理和分子机制(内型)使这一策略得以推进。从这个意义上说,针对重度哮喘儿童的生物疗法变得越来越重要。T2表型在儿童期和青春期最为普遍,而非T2表型通常很少见。本文旨在综述生物药物的作用机制、疗效以及潜在的预测和监测生物标志物,重点关注儿科人群。目前可用的药物有奥马珠单抗、美泊利单抗、贝那利珠单抗、度普利尤单抗和雷泽单抗,美国食品药品监督管理局(FDA)和欧洲药品管理局(EMA)在行政批准处方标准上存在一些差异。此前,我们描述了儿童重度哮喘的特征及其诊断和治疗管理。