What did we learn (and did not learn) from the pediatric direct oral anticoagulant trials, and how might we better design pediatric anticoagulant trials in the future?

Marisol Betensky; Paul Monagle; Christoph Male; Neil A. Goldenberg

doi:10.1016/j.rpth.2023.100140

What did we learn (and did not learn) from the pediatric direct oral anticoagulant trials, and how might we better design pediatric anticoagulant trials in the future?

Marisol Betensky, Paul Monagle, Christoph Male, Neil A. Goldenberg

School of Medicine

Research output: Contribution to journal › Article › peer-review

Abstract

A State of the Art lecture titled “What the direct oral anticoagulants (DOACs) trials did and didn't tell us” was presented at the International Society on Thrombosis and Haemostasis (ISTH) Congress in 2022. The use of DOACs in children is of particular interest as they exhibit several advantages over conventional anticoagulation agents most commonly used in pediatrics. To date, several DOAC pediatric investigational programs (PIPs) have been completed, and although they have provided some of the best quality of evidence in pediatric anticoagulation therapy, the data generated by these trials remain limited. Here, we review and summarize the currently available data provided by the DOAC PIPs, provide perspectives on what we have and have not learned from these trials, and how we might leverage this knowledge to optimize the design of future anticoagulant PIPs. Finally, we summarize relevant new data on this topic presented during the 2022 ISTH Congress.

Original language	English (US)
Article number	100140
Journal	Research and Practice in Thrombosis and Haemostasis
Volume	7
Issue number	3
DOIs	https://doi.org/10.1016/j.rpth.2023.100140
State	Published - Mar 2023

Keywords

anticoagulants
clinical trials
direct-acting oral anticoagulants
pediatrics
thrombosis

ASJC Scopus subject areas

Hematology

Access to Document

10.1016/j.rpth.2023.100140

Cite this

What did we learn (and did not learn) from the pediatric direct oral anticoagulant trials, and how might we better design pediatric anticoagulant trials in the future? / Betensky, Marisol; Monagle, Paul; Male, Christoph et al.
In: Research and Practice in Thrombosis and Haemostasis, Vol. 7, No. 3, 100140, 03.2023.

Research output: Contribution to journal › Article › peer-review

@article{e1feebdc42a5464ca05dd0a84fb19311,

title = "What did we learn (and did not learn) from the pediatric direct oral anticoagulant trials, and how might we better design pediatric anticoagulant trials in the future?",

abstract = "A State of the Art lecture titled “What the direct oral anticoagulants (DOACs) trials did and didn't tell us” was presented at the International Society on Thrombosis and Haemostasis (ISTH) Congress in 2022. The use of DOACs in children is of particular interest as they exhibit several advantages over conventional anticoagulation agents most commonly used in pediatrics. To date, several DOAC pediatric investigational programs (PIPs) have been completed, and although they have provided some of the best quality of evidence in pediatric anticoagulation therapy, the data generated by these trials remain limited. Here, we review and summarize the currently available data provided by the DOAC PIPs, provide perspectives on what we have and have not learned from these trials, and how we might leverage this knowledge to optimize the design of future anticoagulant PIPs. Finally, we summarize relevant new data on this topic presented during the 2022 ISTH Congress.",

keywords = "anticoagulants, clinical trials, direct-acting oral anticoagulants, pediatrics, thrombosis",

author = "Marisol Betensky and Paul Monagle and Christoph Male and Goldenberg, {Neil A.}",

note = "Funding Information: MB has received consultancy fees from Janssen Pharmaceuticals. NAG receives research support and salary support from the National Institutes of Health , National Heart Lung, and Blood Institute for clinical and translational research. He receives or had recently received consultancy fees from Anthos Therapeutics, Bayer, Boehringer-Ingelheim, Chiesi, Daiichi Sankyo, and the University of Colorado-affiliated Academic Research Organization CPC Clinical Research for roles in clinical trial planning or oversight committees (eg, advisory ctte; steering ctte; data and safety monitoring ctte) in pharmaceutical industry-sponsored pediatric clinical trials of antithrombotics. CM receives or had recently received consultancy fees from Anthos Therapeutics, Bayer, Bristol-Myers-Squibb, Chiesi, Janssen Pharmaceuticals, Pfizer, and Norgine. PM has participated on a data safety monitoring board or advisory committee for Bayer and Bristol-Myers-Squibb. Publisher Copyright: {\textcopyright} 2023 The Authors",

year = "2023",

month = mar,

doi = "10.1016/j.rpth.2023.100140",

language = "English (US)",

volume = "7",

journal = "Research and Practice in Thrombosis and Haemostasis",

issn = "2475-0379",