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Rivaroxaban in Paediatric Patients Beyond Clinical Trial Settings - A Real-World Retrospective Cohort Study
Lukas Röthlisberger1,2, Manuela Albisetti1, Alessandra Bosch1
1University Children's Hospital Zürich, Department of Haematology, ZH, Switzerland, Zürich.
Abstract:
BACKGROUND: Direct oral anticoagulants (DOACs) show increasing popularity in the treatment of paediatric venous thromboembolism (VTE), but real-world evidence outside clinical trials remains scarce.
Abstract:
OBJECTIVES: This study aimed to evaluate the use, safety and effectiveness of rivaroxaban in a real-world paediatric cohort at a tertiary care hospital.
Abstract:
METHODS: All children exposed to rivaroxaban between January 2020 and June 2025 at our institution were included into this single-centre retrospective cohort study. In chart reviews, effectiveness was assessed as thrombus recurrence, progression, resolution, or improvement; safety outcomes included clinically relevant bleeding (CRB) and minor bleeding.
Abstract:
RESULTS: Fifty-seven children were included at a median age of 13.8 years (IQR 7.1-15.6); 49% were female. In the treatment group (n = 37), one thrombosis recurred (3%), with thrombus resolution in 19 (54%) and radiologic improvement in 5 patients (14%). In the prophylaxis group (n = 20), no incident VTE occurred. Overall, CRB was rare (7%), all non-major during rivaroxaban exposure and occurred exclusively in children with identifiable additional bleeding risk factors.
Abstract:
CONCLUSIONS: Rivaroxaban demonstrated a favourable effectiveness and safety profile in this real-world paediatric cohort, with outcomes comparable to available real-world and trial data. CRB events clustered in settings with additional bleeding risks, underscoring the importance of patient characteristics in guiding therapy. Our findings support rivaroxaban as a feasible anticoagulation option in children.
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Pharmacovigilance
This process, termed pharmacovigilance, aims to detect, evaluate, and minimize harmful effects related to medication use. The data collection for pharmacovigilance depends on spontaneous reporting systems, where healthcare professionals or patients voluntarily report suspected ADRs.
In some cases, there...