Document is current

Article History

Accepted: 28 April 2021

First Online: 26 May 2021

Declarations

:

: The PERFORM study has received funding from the European Union’s Horizon 2020 Research and Innovation Programme under Grant Agreement No. 668303.

: Roger J. Brüggemann has no conflicts of interest that are directly relevant to the content of this article. Outside of this work, he has served as consultant to and has received unrestricted research grants from Astellas Pharma Inc., F2G, Amplyx, Gilead Sciences, Merck Sharpe and Dohme Corp., and Pfizer Inc. All payments were invoiced by the Radboud University Medical Centre. Saskia N. de Wildt has no conflicts of interest that are directly relevant to the content of this article. Outside of this work she has served as consultant to and had received unrestricted research grant or in kind support from UCB Pharma, Spingotec, and Pfizer Inc. Saskia N. de Wildt is the director of the Dutch Pediatric Formulary and its internationally licensed versions (Stichting Nederlands Kenniscentrum Pharmacotherapy voor Kinderen & Kinderformularium B.V.) All payments were invoiced by the Radboud University Medical Centre. Stan J.F. Hartman, Parth J. Upadhyay, Nienke N. Hagedoorn, Ron A.A. Mathôt, Henriëtte A. Moll, Michiel van der Flier, Michiel F. Schreuder, and Catherijne A. Knibbe have no conflicts of interest that are directly relevant to the content of this article.

: The PERFORM study protocol, and local amendment for the PK sub-study at Erasmus MC, were approved by the local medical ethics review board (CMO Arnhem-Nijmegen, NL58103.091.16, 2016-3085). The medical ethics review board waived the need for formal ethics approval for the POPSICLE study according to the Dutch Law on Human Research (REB 2016-2689).

: Informed or deferred consent was provided by all parents/caregivers with the consent/assent of the child depending on the child’s age and/or capabilities.

: Not applicable.

: Data and material are available upon request with the corresponding author.

: The model code is provided in the ESM.

: Development of the POPSICLE research protocol (SH, RB, MvdF, MS, SdW), development of the PERFORM sub-study protocol amendment (NH, MvdF, HM, SdW, SH), including patients and collection of samples from the POPSICLE study (SH), including patients and collecting samples from the PERFORM study (NH), bioanalysis of samples (RM), data analysis and pop-PK modeling (SH, PU, CK, SdW), writing first version of manuscript (SH, PU, CK, SdW), and completing final version of manuscript (SH, PU, NH, RM, HM, MvdF, MS, RB, CK, SdW).