title: The need for precision medicine clinical trials in childhood asthma: rationale and design of the PUFFIN trial.
abstract: 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.
authors.affiliation: Department of Respiratory Medicine, Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands.
authors.forename: Susanne Jh
authors.initials: SJ
authors.lastname: Vijverberg
authors.affiliation: Department of Pediatrics, Pediatric Pulmonology & Allergology, Erasmus Medical Center-Sophia Children's Hospital, Rotterdam, The Netherlands.
authors.forename: Mariëlle W
authors.initials: MW
authors.lastname: Pijnenburg
authors.affiliation: Division of Pharmacoepidemiology & Clinical Pharmacology, Utrecht Institute for Pharmaceutical Science, Utrecht University, Utrecht, The Netherlands.
authors.forename: Anke M
authors.initials: AM
authors.lastname: Hövels
authors.affiliation: University of Groningen, University Medical Center Groningen, Department of Pediatric Pulmonology & Pediatric Allergology, Beatrix Children's Hospital, Groningen, The Netherlands.|University of Groningen, University Medical Center Groningen, Groningen Research Institute for Asthma & COPD (GRIAC), Groningen, The Netherlands.
authors.forename: Gerard H
authors.initials: GH
authors.lastname: Koppelman
authors.affiliation: Department of Respiratory Medicine, Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands.
authors.forename: Anke-Hilse
authors.initials: AH
authors.lastname: Maitland-van der Zee
mesh_terms: D001249:Asthma / Q000453:epidemiology / Q000235:genetics / Q000628:therapy*; D002648:Child; D004311:Double-Blind Method; D006801:Humans; D015337:Multicenter Studies as Topic / Q000379:methods*; D057285:Precision Medicine / Q000379:methods*; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D018343:Receptors, Adrenergic, beta-2 / Q000235:genetics
chemical_list: C553443:ADRB2 protein, human; D018343:Receptors, Adrenergic, beta-2