Strong Efficacy and Safety Profile Rivaroxaban Demonstrated by in phase III Study in Children with thromboembolism

Results from the phase III EINSTEIN-Jr. study showed a strong efficacy and safety profile of rivaroxaban in children with VTE, with results similar to those seen in previous studies in adults. Recurrent venous thromboembolism occurred at a low incidence with a numerical reduction of events in children treated with rivaroxaban compared with standard of care (heparins alone or in combination with a vitamin K antagonist such as warfarin). Major bleeding was not observed in the rivaroxaban group. Children received rivaroxaban as tablets or as a newly-developed suspension for oral use. The findings from EINSTEIN-Jr. were presented as a late breaker at the 27th International Society on Thrombosis and Haemostasis Congress (ISTH) in Melbourne, Australia today.

“Current treatment of VTE in children often relies on parenteral administration of anticoagulants and requires laboratory monitoring and dose adjustments. Up to now, pediatric anticoagulant treatment regimens were based mostly on observational data and on extrapolation of data obtained in adults. The EINSTEIN-Jr. study represents an important step forwards for the treatment of VTE in children,” said Professor Christoph Male, from the Department of Pediatrics, Medical University of Vienna in Austria.

The study showed that 4 out of 335 (1.2%) children treated with rivaroxaban and 5 out of 165 (3.0%) treated with standard of care had recurrent VTE. Furthermore, repeat imaging carried out at the end of the treatment period demonstrated an improved effect of rivaroxaban on thrombotic burden as compared with standard of care. Clinically relevant bleeding occurred in 10 children (3.0%; all were non-major bleeds) treated with rivaroxaban and in 3 children (1.9%; two were major bleeds and one was a non-major bleed) treated with standard of care.The absolute and relative safety and efficacy results seen in the EINSTEIN-Jr. study are consistent to those from previous rivaroxaban studies in adults.

“We have demonstrated that the use of the pediatric body weight-adjusted rivaroxaban regimens is efficacious and has shown positive safety results for the treatment of VTE in children,” said Professor Male. “Availability of the rivaroxaban suspension for oral use will obviate the need for manipulations of adult dosage forms and substantially reduce the number of injections needed for standard anticoagulation treatment and blood sampling. The pediatric rivaroxaban regimen will represent an advantageous alternative treatment for children with VTE in the future. The data are consistent with the clinical outcomes observed in adult patients, as well as in other at risk subgroups of patients in the EINSTEIN program.”

“Today, venous thromboembolism is being identified more often in childhood than in the past due to the increased survival of children with life-threatening or chronic medical conditions and also increased awareness of VTE among pediatricians,” said Dr Joerg Moeller, Member of the Executive Committee at Bayer AG’s Pharmaceuticals Division and Head of Research and Development. “The study results bring us one-step closer to providing pediatricians with a new therapeutic option for children. Rivaroxaban has the most extensive clinical study program of any non-vitamin K antagonist oral anticoagulant (NOAC) and we continue to investigate which patients will benefit most from use of this product.”

Bayer will submit an application to the European Medicines Agency (EMA) for the extension of the Xarelto marketing authorization to make this new treatment option, including the granules for oral suspension, available for children with venous thromboembolism.

VTE includes cerebral vein and sinus thrombosis, a blood clot in the brain, pulmonary embolism (PE), a blood clot that travels to the lung, and deep vein thrombosis (DVT), a blood clot in a deep vein (often in the legs). VTE is an increasingly common complication among hospitalized children with the most common risk factor for VTE being venous catheterization. Currently recommended treatment options for VTE include unfractionated heparin, low molecular weight heparin, fondaparinux with or without vitamin K antagonist therapy. No non-vitamin K oral anticoagulant is currently approved for use in this setting.

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