Direct oral anticoagulants: What will be their role in children?

Abstract

Thrombotic events in children differ from those in adults in epidemiology, pathophysiology, and anatomical location. However, anticoagulation in children is mostly based on evidence from adults while scarce evidence exists from children. The classical anticoagulants currently used in children have several limitations, resulting in the need for regular monitoring. Several direct oral anticoagulants (DOACs) are now authorized for adults in whom they have established efficacy and safety without the need for monitoring. Given their pharmacological properties and the special characteristics of children requiring anticoagulation, the DOACs have the potential to be particularly suitable for children. All currently approved DOACs have paediatric development plans, targeting various indications for prevention and treatment of thrombosis. Paediatric formulations are being developed and systematic age-specific dosing information will be generated. Whether therapeutic drug monitoring will be necessary in certain situations in children remains to be elucidated. The results of ongoing clinical studies still need to demonstrate whether there is a positive benefit-risk balance in all targeted paediatric indications and age-groups, particularly in indications that have not been explored in adults, such as catheter-related thrombosis or congenital heart disease. If the advantages of DOACs bear out in the results of the current paediatric studies, they will likely be used widely in children. As of now, the DOACs should not be used routinely in children as there is still insufficient information on appropriate dosing, safety and efficacy. The paediatric community is encouraged to promote participation of children and adolescents into the multiple ongoing studies of DOACs.

Keywords: Children; Direct oral anticoagulants; Dose; Drug development; Monitoring; Thrombosis.