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Use of Direct Oral Anticoagulants in Children and Adolescents
1Division of Hematology and Children's Research Center, University Children's Hospital, Zurich, Switzerland.
Insights
Direct oral anticoagulants (DOACs) show promise for pediatric anticoagulation, offering a safer and more effective alternative to traditional treatments like low-molecular-weight heparin (LMWH) and vitamin K antagonists (VKA) in children.
Area of Science:
- Pediatric Hematology
- Pharmacology
- Thrombosis Research
Background:
- Current pediatric anticoagulation relies on adult-extrapolated regimens for low-molecular-weight heparin (LMWH) and vitamin K antagonists (VKA), which are suboptimal due to unique pediatric hemostatic system characteristics.
- Existing anticoagulants present significant disadvantages, particularly for newborns and infants, complicating optimal treatment.
- Direct oral anticoagulants (DOACs) have emerged as effective alternatives in adults, prompting investigation into their pediatric use.
Purpose of the Study:
- To review available data from terminated and ongoing controlled trials on direct oral anticoagulants (DOACs) in pediatric and adolescent populations.
- To evaluate the efficacy and safety of DOACs compared to traditional anticoagulants in children.
- To summarize findings on pediatric-specific formulations and dosing guidelines for DOACs.
Main Methods:
- Review of terminated and ongoing controlled clinical trials involving direct oral anticoagulants (DOACs) in children and adolescents.
- Analysis of data from phase III trials assessing DOACs for treatment and prevention of thrombotic events.
- Evaluation of safety and efficacy profiles of DOACs in pediatric patients with diverse clinical conditions.
Main Results:
- First results from phase III pediatric trials indicate that DOACs are comparable in efficacy and safety to LMWH and VKA.
- DOACs demonstrate potential to overcome the limitations of traditional anticoagulants in pediatric patients, including newborns and infants.
- Ongoing trials are establishing age- and weight-adjusted dosing guidelines and pediatric-specific formulations for DOACs.
Conclusions:
- Direct oral anticoagulants (DOACs) represent a promising advancement in pediatric anticoagulation, offering improved safety and efficacy.
- DOACs are poised to become a viable alternative to LMWH and VKA for managing thrombotic events in children.
- Further research and ongoing trials are crucial for optimizing DOAC therapy in pediatric populations.
Abstract:
While the need for anticoagulation in children has increased over the last decades, dose regimens of currently used anticoagulants, including low-molecular-weight heparin (LMWH) and vitamin K antagonist (VKA), are still extrapolated from adult guidelines because well-designed clinical trials were never performed in children. This approach is not optimal due to specific pediatric features of the hemostatic system and pathophysiology of thrombosis. These anticoagulants also present several disadvantages that further hamper optimal anticoagulation of pediatric patients, especially newborns and infants. The new direct oral anticoagulants (DOACs), which have the potential to overcome these disadvantages, were extensively investigated in adults and have become a valid alternative to LMWH and VKA for anticoagulation in the adult population. Several pediatric trials on all approved DOACs are currently ongoing, providing specific pediatric formulations and age- and weight-adjusted dose guidelines. First results of phase III trials indicate that DOACs are at least as efficient and safe as LMWH and VKA for the treatment and prevention of thrombotic events in children with different clinical conditions. This review article summarizes available data from terminated and ongoing controlled trials on DOACs in children and adolescents.
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