Long-term study confirms safety and effectiveness of rivaroxaban for children

Venous thromboembolism (VTE) is a life-threatening complication in children with serious underlying conditions such as heart defects or cancer. Treatment or prevention of thrombosis poses an additional challenge in everyday clinical practice. A therapy using the active ingredient rivaroxaban specifically tailored to children was successfully tested for the first time in 2020. Now, long-term data confirm the benefits of this drug treatment even for extended use. The study was conducted by an international research team led by MedUni Vienna and has been published in the journal "The Lancet Haematology".

The long-term data collected by the research team led by Christoph Male from the Department of Pediatrics and Adolescent Medicine at MedUni Vienna provides the first reliable evidence for extended anticoagulation in children. The study assessed extended treatment in a cohort of around 500 children and adolescents from the EINSTEIN Jr study, whose data on acute phase anticoagulation was already published in 2020. Those study results, also published under the leadership of MedUni Vienna, showed that the anticoagulant rivaroxaban is at least as effective and safe as the standard anticoagulants used to date in children with venous thromboembolism, but also offers a number of advantages for young patients. The positive results paved the way for the worldwide approval of rivaroxaban for children in 2021. Until now, there has been a lack of research on extended anticoagulation in children with VTE, and with rivaroxaban in particular – a gap now closed by the recently published long-term study: The research shows that even with long-term treatment of up to one year, there is only a low risk of VTE recurrence and serious bleeding. Rivaroxaban is therefore the first scientifically proven age-appropriate alternative to the standard anticoagulation therapies available for children up to now.

Specifically adapted to children

Venous thromboembolism (VTE) occurs when blood clots form in deep veins causing local vessel obstruction or embolization of the clot to the lungs – a potentially life-threatening condition. While this condition has been well researched in adults, there has long been a lack of solid data for children. Until recently, treatment with anticoagulant drugs was based on the off-label use of drugs that were originally developed for adults, such as heparin or vitamin K antagonists. These drugs have some disadvantages that are particularly problematic for children, such as administration by injection and the need for regular blood tests. Direct oral anticoagulants such as rivaroxaban, which have several advantages in practical use, were also originally developed for adults, but in recent years have been specifically adapted for children and tested in clinical trials.

Our EINSTEIN Jr. study and the long-term investigation now available show that rivaroxaban is an effective and safe option for preventing recurrent thrombosis in children, not only during for acute treatment but also extended treatment – and thus offers the first scientifically sound, age-appropriate alternative to existing standard therapies."

Christoph Male, study leader 

The importance of the drug in anticoagulation in children is also highlighted in an accompanying editorial in The Lancet Haematology.