OC 15.5 - Validating Direct Oral Anticoagulants (DOAC) for Use in Children by the Throm-PED DOAC Registry of the International Pediatric Thrombosis Network

Sunday, July 10, 2022

11:45 AM – 12:00 PM

Location: ExCel Center, ICC Capital Suite Room 14-16

Presentation Format: In-Person

Presenting Author(s)

SH

Susanne Holzhauer

MD
Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie, Universitätsmedizin Berlin.
Berlin, Berlin, Germany

Co-Author(s)

Paul Monagle, MD

Professor
Haematology Research, Murdoch Children's Research Institute
Melbourne, Victoria, Australia
Christoph Male, MD, MSc

Head of the Pediatric Hemostasis & Thrombosis Unit
Department of Paediatrics, Medical University of Vienna, Vienna, Austria.
Vienna, Wien, Austria
Rukhmi Bhat, MD,MS, DNB

Associate Professor of Pediatrics (Hematology, Oncology, and Stem Cell Transplantation)
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, United States
MB

Mohammad Bordbar

Pediatric Hematologist-Oncologist
Shiraz University
Chiraz, Fars, Iran
Rungrote Natesirinilkul, MD

Associate Professor
Department of Pediatrics, Faculty of Medicine, Chiang Mai University, Chiang Mai, Thailand
Chiang Mai, Chiang Mai, Thailand
SS

Surbhi Saini

Assistant Professor of Pediatrics, Hematology and Oncology
Department of Pediatrics, Washington University in St. Louis
St. Louis, Missouri, United States
MS

Mindy L. Simpson

Pediatric Hematologist-Oncologist
Rush University medical Center
chicago, Illinois, United States
C. Heleen Van Ommen, MD, PhD

Pediatric Hematologist
Erasmus MC Sophia Children's Hospital
Rotterdam, Zuid-Holland, Netherlands
LR

Leslie Raffini

Associate Professor of Pediatrics
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States

Background: Dabigatran and rivaroxaban have recently been approved for treatment of venous thromboembolism in children. Based on the risk benefit profiles published so far, we expect DOACs to be widely used in children. The strict inclusion criteria for participation in the clinical trials limit their generalizability, particularly for those with serious medical conditions that account for a significant proportion of pediatric VTE patients in clinical practice.

Aims: To study efficacy and safety of DOACs in a large heterogeneous pediatric population. To expand our knowledge on treatment strategies and outcomes in children across different risk profiles and comorbidities including cancer and renal disease.

Methods: An international, multicentre, prospective observational cohort study of patients < 18 years with thromboembolic disease treated with DOACs. Data collection includes thrombosis location, risk factors, medical conditions and concurrent medications. Outcomes (thrombus progression/recurrence and bleeding) are assessed every 3 months for 12 months. Additional variables include adherence, drug levels, renal function and dose adjustments.

Results: A total of 102 patients from 9 centers have been included in the registry to date, of which 81% of patients were >12 years old (Figure 1) and most 79% received rivaroxaban. Pulmonary embolism and thrombosis of the lower extremities were more common (30% each, followed by cerebral thrombosis (16%). Risk factors included cancer (13%) and renal disease (5%). Of the 39 patients with follow-up at 3 months, bleeding was reported in 3 (8%) patients and thrombosis progression in 2 (6%) (Table 1).

Conclusion(s): Real world data collection is essential to assess the benefit risk profile of DOACs in children, to identify specific patient groups at risk of worse outcome and to understand drug interactions and the need for dose adjustments. Further data on younger age groups and children with comorbidities are required.