Abstract Number: OC 15.5
Meeting: ISTH 2022 Congress
Background: Dabigatran and rivaroxaban have recently been approved for treatment of venous thromboembolism in children. Based on the risk benefit profiles published so far, we expect DOACs to be widely used in children. The strict inclusion criteria for participation in the clinical trials limit their generalizability, particularly for those with serious medical conditions that account for a significant proportion of pediatric VTE patients in clinical practice.
Aims: To study efficacy and safety of DOACs in a large heterogeneous pediatric population. To expand our knowledge on treatment strategies and outcomes in children across different risk profiles and comorbidities including cancer and renal disease.
Methods: An international, multicentre, prospective observational cohort study of patients < 18 years with thromboembolic disease treated with DOACs. Data collection includes thrombosis location, risk factors, medical conditions and concurrent medications. Outcomes (thrombus progression/recurrence and bleeding) are assessed every 3 months for 12 months. Additional variables include adherence, drug levels, renal function and dose adjustments.
Results: A total of 102 patients from 9 centers have been included in the registry to date, of which 81% of patients were >12 years old (Figure 1) and most 79% received rivaroxaban. Pulmonary embolism and thrombosis of the lower extremities were more common (30% each, followed by cerebral thrombosis (16%). Risk factors included cancer (13%) and renal disease (5%). Of the 39 patients with follow-up at 3 months, bleeding was reported in 3 (8%) patients and thrombosis progression in 2 (6%) (Table 1).
Conclusion(s): Real world data collection is essential to assess the benefit risk profile of DOACs in children, to identify specific patient groups at risk of worse outcome and to understand drug interactions and the need for dose adjustments. Further data on younger age groups and children with comorbidities are required.
To cite this abstract in AMA style:
Holzhauer S, Monagle P, Male C, Bhat R, Bordbar M, Natesirinilkul R, Saini S, Simpson M, van Ommen C, Raffini L. Validating Direct Oral Anticoagulants (DOAC) for Use in Children by the Throm-PED DOAC Registry of the International Pediatric Thrombosis Network [abstract]. https://abstracts.isth.org/abstract/validating-direct-oral-anticoagulants-doac-for-use-in-children-by-the-throm-ped-doac-registry-of-the-international-pediatric-thrombosis-network/. Accessed March 21, 2024.« Back to ISTH 2022 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/validating-direct-oral-anticoagulants-doac-for-use-in-children-by-the-throm-ped-doac-registry-of-the-international-pediatric-thrombosis-network/