1 - Hospital Dona Estefânia, Unidade Local de Saúde de São José, Paediatric Nephrology Unit, Lisbon, Portugal
2 - Hospital Dona Estefânia, Unidade Local de Saúde de São José, Paediatric Haematology Unit, Lisbon, Portugal
- 56th Annual Meeting of the European Society for Paediatric Nephrology, 24-26/9/2024, Valência, Espanha, Poster
- Resumo publicado em: Pediatr Nephrol 2024;39(Suppl 1):S272.
Abstract:
Purpose: Nephrotic syndrome (NS) is associated with a multifactorial hypercoagulable state. Congenital NS (CNS) exhibits a higher prevalence of thrombotic events compared to other types. Direct oral anticoagulants (DOAC) have been approved for paediatric acute venous thromboembolism. We present 2 CNS paediatric cases treated with rivaroxaban.
Methods: Rivaroxaban was used for treatment (Case 1) and prophylaxis (Case 2) of thrombotic events in CNS, in the absence of contraindications (antiphospholipid syndrome; risk of bleeding; liver dysfunction). Therapeutic doses were determined per the latest guidelines.
Results:
Case 1: A 2-month-old male previously diagnosed with CNS with homozygous mutation in the NPHS1 gene, underwent central venous catheter (CVC) replacement during which multiple thrombi were seen. In the first 24 hours, the patient developed clinical signs compatible with pulmonary embolism (acute pallor, tachycardia, shortness of breath, hypoxemia, without fever). Chest radiograph showed a peripheral condensation on the left hemithorax. CT-angiography scan ruled out a major pulmonary embolism, with inconclusive result for peripheral embolism. Despite therapeutic doses of enoxaparin, adjustments were difficult with persistently low anti-Xa levels. The switch to rivaroxaban was performed after 11 days, and doses were regularly adjusted based on patient’s weight. No adverse or other thrombotic events were reported, despite maintaining CVC. As expected, chronic kidney disease progressed (eGFR of 22 mL/min/1.73m2) at 19 months and rivaroxaban was suspended.
Case 2: A 8-month-old female with CNS and heterozygous mutations in the NHPS1 gene, underwent multiple CVC replacements due to recurrent obstruction despite regular heparinisation and alteplase administrations. Although there were no systemic thrombotic episodes, considering the high risk of thrombosis, prophylaxis with rivaroxaban was initiated. At the time, the patient presented an eGFR of 54 mL/min/1.73m2 (1-2 SD below expected eGFR). Weight-adjusted dose was prescribed. No severe adverse or thrombotic events reported until now, with 19 months. She still has a CVC with only one readmission due to its obstruction.
Conclusion: These cases suggest that the safety and efficacy profile of rivaroxaban may be encouraging for treating and preventing venous thromboembolism in CNS. However, additional studies are warranted to optimize DOAC use in children with complex conditions, such as CNS, allowing for more tailored management of anticoagulation in this high-risk population.
Keywords: congenital nephrotic syndrome, rivaroxaban, venous thromboembolism