Vijverberg Susanne Jh, Pijnenburg Mariëlle W, Hövels Anke M, Koppelman Gerard H, Maitland-van der Zee Anke-Hilse
Department of Respiratory Medicine, Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands.
Department of Pediatrics, Pediatric Pulmonology & Allergology, Erasmus Medical Center-Sophia Children's Hospital, Rotterdam, The Netherlands.
Pharmacogenomics. 2017 Mar;18(4):393-401. doi: 10.2217/pgs-2016-0174. Epub 2017 Feb 22.
A 'one-size fits all'-approach does not fit all pediatric asthma patients. Current evidence suggests that in children with persistent asthma, ADRB2 genotype-guided treatment can improve treatment outcomes, yet this evidence is mainly derived from observational and genotype-stratified studies. Implementation of precision medicine-guided asthma treatment in clinical practice will only occur if randomized clinical trials can show that this approach will improve patient outcomes and is cost effective. In this paper, we will discuss why precision medicine trials are currently needed to improve childhood asthma management and present the rationale and design of the PUFFIN trial, that has been set up to address this need.
“一刀切”的方法并不适用于所有儿童哮喘患者。目前的证据表明，对于持续性哮喘患儿，基于β2肾上腺素能受体（ADRB2）基因型的指导治疗可改善治疗效果，但这一证据主要来自观察性研究和基因型分层研究。只有当随机临床试验能够证明这种方法可改善患者预后且具有成本效益时，精准医学指导的哮喘治疗才会在临床实践中得以实施。在本文中，我们将讨论为何目前需要精准医学试验来改善儿童哮喘管理，并介绍为满足这一需求而设立的PUFFIN试验的基本原理和设计。
Zotero 插件