The need for precision medicine clinical trials in childhood asthma: rationale and design of the PUFFIN trial

Susanne Jh Vijverberg; Mariëlle W Pijnenburg; Anke M Hövels; Gerard H Koppelman; Anke-Hilse Maitland-van der Zee

doi:10.2217/pgs-2016-0174

The need for precision medicine clinical trials in childhood asthma: rationale and design of the PUFFIN trial

Susanne Jh Vijverberg, Mariëlle W Pijnenburg, Anke M Hövels, Gerard H Koppelman, Anke-Hilse Maitland-van der Zee^*

^*Corresponding author for this work

Academic Medical Center
Department of Pediatrics, Pediatric Pulmonology & Allergology, Erasmus Medical Center-Sophia Children's Hospital, Rotterdam, The Netherlands.
University of Groningen, University Medical Center Groningen, Department of Paediatric Pulmonology and Paediatric Allergology, Beatrix Children's Hospital, Groningen, The Netherlands; University of Groningen, University Medical Center Groningen, Groningen Research Institute for Asthma and COPD (GRIAC), Groningen, The Netherlands.

Research output: Contribution to journal › Review article › peer-review

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.

Original language	English
Pages (from-to)	393-401
Number of pages	9
Journal	Pharmacogenomics
Volume	18
Issue number	4
DOIs	https://doi.org/10.2217/pgs-2016-0174
Publication status	Published - Mar 2017

Keywords

Asthma
Child
Double-Blind Method
Humans
Multicenter Studies as Topic
Precision Medicine
Randomized Controlled Trials as Topic
Receptors, Adrenergic, beta-2
Journal Article
Review

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The Need for Precision Medicine Clinical Trials in Childhood Asthma Rationale and Design of the Puffin TrialFinal published version, 1.32 MBLicence: Taverne

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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.",

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AU - Maitland-van der Zee, Anke-Hilse

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KW - Child

KW - Double-Blind Method

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KW - Multicenter Studies as Topic

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KW - Randomized Controlled Trials as Topic

KW - Receptors, Adrenergic, beta-2

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The need for precision medicine clinical trials in childhood asthma: rationale and design of the PUFFIN trial

Abstract

Keywords

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