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?

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.