Real world experience of direct oral anticoagulant (DOAC) use in Australian children.

BACKGROUND

Direct oral anticoagulant (DOAC) use in children is increasing, supported by multi-center randomized controlled trials (RCTs) and registry data. This study describes real-world experience using DOACs in a paediatric population.

METHODS

We performed a retrospective case series at a single Australian paediatric tertiary hospital, including children aged 0-18 years treated with a DOAC for thrombosis prophylaxis or treatment between January 1st 2021 to May 1st 2025. Data was obtained through retrospective review of patient medical records. Patients were excluded if their medical records lacked sufficient documentation to allow for our analysis. Primary outcomes measured were thrombus resolution, recurrence, extension or development. Principal safety outcomes measured were major or clinically relevant non-major bleeding (CRNMB) and adverse events. Qualitative data on quality of life (QoL) was also obtained if available.

FINDINGS: Out of 69 patients prescribed a DOAC, 67 met inclusion criteria creating 74 treatment episodes. Two patients were excluded due to insufficient documentation. Indications for treatment included therapeutic (n = 49), primary prophylaxis (n = 4), secondary prophylaxis (n = 10), central venous line (CVL) prophylaxis for patients on longterm total parenteral nutrition (TPN) (n = 5) and localized intravascular coagulopathy (LIC) secondary to venous malformation (n = 6). Treatment failure occurred in four episodes (5%), with one episode in each of the therapeutic, primary prophylaxis, secondary prophylaxis, and LIC groups. There were no episodes of fatal thromboembolism. There were no major bleeding events or treatment-related deaths, three (4%) CRNMB events and four (5%) DOAC related side effects.

INTERPRETATION: We confirm the effectiveness of DOACs for the prophylaxis and treatment of paediatric thromboembolism and report acceptable risk of bleeding and side effects.