Abstract
Venous thromboembolism (VTE) in children can lead to significant morbidity and mortality.
Traditionally, treatment for thrombotic events in pediatric patients has been limited
mainly to unfractionated heparin, low-molecular-weight heparin (LMWH), or vitamin
K antagonists. Since the first non–vitamin K antagonist oral anticoagulant (NOAC)
was approved for adult use, these agents have gained popularity for a variety of indications.
This is largely due to their ease of administration, favorable pharmacokinetic and
pharmacodynamic profile, decreased food interactions, and decreased need for therapeutic
drug monitoring. Treating and preventing VTE with traditional anticoagulants in pediatric
patients presents many challenges. This systematic review evaluated the current literature
regarding pediatric NOAC trials. Additionally, based on an up-to-date query of clinicaltrials.gov , we detail current ongoing and as-yet unpublished clinical trials, study outcomes,
and projected completion dates. Published pediatric NOAC trials have included 1,007
total children to date and have ranged from phase 1 to 4, with “indications” including
both thromboembolism prophylaxis and VTE treatment. Three recent phase 3 trials, specifically
involving rivaroxaban and dabigatran, have shown the agents to be at least as effective
as traditional anticoagulants for acute and/or extended VTE treatment, with low frequency
of recurrent thrombosis and clinically significant bleeding rates. Additionally, specially
developed and tested pediatric formulations have allowed for accurate and reliable
dosing, oral administration, stable pharmacokinetics and pharmacodynamics, and fewer
drug or food interactions. Ongoing trials, anticipated for completion in the next
few years, will reveal important information with regard to thromboembolism prophylaxis
in special pediatric subpopulations and settings.
Keywords pediatric VTE - thromboprophylaxis - NOACs
