Abstract Body: Introduction: Pediatric arterial ischemic stroke (AIS) affects 1-2 per 100,000 children. Although rare, mortality and morbidity is significant with approximately 10% recurrence rate within the first year. Despite this, evidence-based treatment and prevention guidelines of pediatric AIS remain insufficient. Direct oral anticoagulants (DOACs) are approved in many countries for the treatment and prevention of pediatric venous thrombosis but its use in pediatric arterial thrombosis is less established. This study is the first to evaluate the off-label use of rivaroxaban in pediatric AIS.
Methods: A retrospective chart review was approved by Cook Children’s Medical Center (CCMC) institutional review board. Patients (0 to <18 years) treated at CCMC for acute AIS that received rivaroxaban between June 21, 2021-April 1, 2024 were reviewed. Patients treated for concomitant venous thrombosis and/or without follow up imaging were excluded. Data was de-identified and SAS 8.3 was used for analysis. Given small sample, data were summarized using descriptive statistics.
Results: Thirty-two patients were reviewed and 14 met eligibility criteria. Patient demographics and medical history are summarized in Table 1. All had acute AIS confirmed via brain magnetic resonance imaging (MRI), with most strokes affecting the anterior circulation (8, 57.1%) and with arteriopathy as the most common stroke etiology (10, 71.4%) (Table 2). Thirteen (92.9%) received antithrombotic therapy (heparin, enoxaparin, acetylsalicylic acid) prior to DOAC initiation and all 14 were given rivaroxaban afterwards. At the time of data analysis, patients had a median of 4 months of DOAC therapy (interquartile range: 3-7). Four patients remain on treatment (28.6%), 7 completed treatment (50.0%), 1 stopped due to the rivaroxaban shortage (7.1%), 1 discontinued due to bleeding complications (concomitant antiplatelet therapy) (7.1%), and 1 discontinued due to treatment failure (7.1%). All other adverse events were minor not requiring treatment discontinuation (Table 3).
Conclusion: In this study, 14 pediatric AIS patients were treated with rivaroxaban. Only one had it discontinued for patient safety but concomitant therapy with acetylsalicylic acid, made rivaroxaban causality inconclusive. The only treatment failure in our series was a patient with bow hunter syndrome. Even though this study suggests rivaroxaban might be a safe and effective therapeutic option in pediatric AIS, additional research is needed.