Associate Professor Weill Cornell Medicine New York, New York, United States
Background: Myeloproliferative neoplasms (MPNs) are rare disorders associated with thrombotic and hemorrhagic complications. Patients who develop thrombotic events are often put on long-term anticoagulation, yet there is a risk of bleeding in these patients due to various factors such as acquired von Willebrand’s disease (aVWD) and vascular changes. Therefore, it is important to evaluate any bleeding complications in patients treated with anticoagulation.
Aims: To describe the use of anticoagulation and bleeding complications in children with MPNs .
Methods: Patients diagnosed with an MPN between 0-21 years old were enrolled on an observational, IRB-approved study. Charts were reviewed to identify those with a history of venous thromboembolism (VTE), and from their charts, demographic information, medication history, blood tests, and clinical events were recorded.
Results: In a cohort of 47 pediatric MPN patients, 6 patients were identified to have VTE. All six patients were female. Four patients had a JAK2 mutation, one patient had an MPL mutation, and one patient was negative for known driver mutations. Among VTE types, 4 patients had Budd Chiari syndrome, and 2 patients had cerebral venous sinus thromboses (Table#1). All patients were treated at some point with anticoagulant therapy for management of their VTE events. Anticoagulants used included apixaban, rivaroxaban, warfarin, enoxaparin, and fondaparinux. One patient had a CNS bleeding event while on enoxaparin (and did not have aVWD at the time), while no other patients experienced significant bleeding.
Conclusion(s): In this cohort of pediatric MPN patients, 12.7% of children required anticoagulation for a VTE. Only one of those patients had a significant bleeding event on anticoagulation, and patients on direct oral anticoagulants did not have bleeding. Anticoagulation can be a safe and important part of the treatment of children with MPNs and VTE and more study is needed to evaluate bleeding risk in this population.
.jpeg.jpg "Nicole Kucine, MD, MS (she/her/hers) photo")