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Published on: February 10, 2023
Treatment of venous thromboembolism in pediatric patients
1Department of Pediatrics, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.
Insights
Pediatric venous thromboembolism (VTE) is a growing concern in children with medical conditions. New direct oral anticoagulants show promise for improving VTE treatment in this vulnerable population.
Area of Science:
- Pediatric Hematology
- Thrombosis Research
- Clinical Pharmacology
Background:
- Venous thromboembolism (VTE) is uncommon in healthy children but increasingly prevalent in those with underlying medical conditions.
- Pediatric VTE cases are diverse, varying by age, thrombosis site, and comorbidities, complicating treatment strategies.
- Current treatment relies heavily on adult data and expert consensus due to a lack of pediatric-specific clinical trials.
Purpose of the Study:
- To review the current landscape of pediatric venous thromboembolism (VTE) management.
- To highlight challenges unique to pediatric anticoagulation, including developmental hemostasis and central venous access devices.
- To discuss emerging therapies and the role of collaborative research in advancing pediatric VTE care.
Main Methods:
- Review of existing literature and clinical trial data on pediatric VTE.
- Analysis of treatment challenges, including drug formulations and monitoring for neonates and infants.
- Discussion of ongoing randomized clinical trials for direct oral anticoagulants in pediatric VTE.
Main Results:
- Existing anticoagulants (low-molecular-weight heparin, warfarin) have limitations like injections and frequent monitoring.
- Direct oral anticoagulants (DOACs) are under investigation, with trials expected to yield significant new evidence.
- International registries are crucial for studying rare pediatric VTE subgroups and guiding disease-specific therapies.
Conclusions:
- Pediatric VTE treatment is challenged by a lack of evidence and unique patient factors.
- Ongoing trials of DOACs offer potential for improved pediatric VTE management.
- Collaborative research and registries are essential for advancing personalized VTE therapy in children.
Abstract:
Venous thromboembolism (VTE) is rare in healthy children, but is an increasing problem in children with underlying medical conditions. Pediatric VTE encompasses a highly heterogenous population, with variation in age, thrombosis location, and underlying medical comorbidities. Evidence from pediatric clinical trials to guide treatment of VTE is lacking so treatment is often extrapolated from adult trials and expert consensus opinion. Aspects unique to children include developmental hemostasis and the major role of central venous access devices. There is an absence of information regarding the optimal target levels of anticoagulation for neonates and infants and lack of suitable drug formulations. Anticoagulants, primarily low-molecular-weight heparin and warfarin, are used to treat children with symptomatic VTE. These drugs have significant limitations, including the need for subcutaneous injections and frequent monitoring. Randomized clinical trials of direct oral anticoagulants in pediatric VTE are ongoing, with results anticipated soon. These trials will provide new evidence and options for therapy that have the potential to improve care. International collaborative registries offer the ability to study outcomes of rare subgroups of pediatric VTE (eg, renal vein thrombosis), and will be important to ultimately guide therapy in a more disease-specific manner.
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