PB0276 - Real World Data of Pediatric Thromboembolic Disease by the International Pediatric Thrombosis Network

Sunday, July 10, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

C. Heleen Van Ommen, MD, PhD

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

Co-Author(s)

MA

Manuela Albisetti

Head of Hemophilia
Hematology Department, University Children’s Hospital, Zurich, Switzerland
Zürich, Zurich, Switzerland
MB

Mihir D. Bhatt, MD

Pediatric Hematologist
McMaster Children’s Hospital
Hamilton, Ontario, Canada
BB

Brian Branchford

Pediatric Hematologist-Oncologist
Versiti Medical Sciences Institute
Wauwatosa, Wisconsin, United States
Anthony K. Chan, MBBS, FRCP (C), MBBS

Pediatric Hematologist
Department of Pediatrics, McMaster Children's Hospital
Hamilton, Ontario, Canada
Neil A. Goldenberg, MD, PhD

Professor
Institute for Clinical and Translational Research, Johns Hopkins All Children's Hospital, St. Petersburg, USA
Saint Petersburg, Florida, United States
SH

Susanne Holzhauer

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

Head of the Pediatric Hemostasis & Thrombosis Unit
Department of Paediatrics, Medical University of Vienna, Vienna, Austria.
Vienna, Wien, Austria
Paul Monagle, MD

Professor
Haematology Research, Murdoch Children's Research Institute
Melbourne, Victoria, Australia
UN

Ulrike Nowak-Gottl

Pediatric Hematologist
University Hospital Schleswig-Holstein
Kiel, Hamburg, Germany
SR

Shoshana Revel-Vilk

Pediatric Hematologist
Shaare Zedek Medical Center
Jerusalem, Yerushalayim, Israel
GS

Gabriela B. Sciuccati, MD

Pediatric Hematologist
Hospital de Pediatría "Prof. Dr. J.P.Garrahan"
CABA, Ciudad Autonoma de Buenos Aires, Argentina
Nongnuch Sirachainan, MD

Professor
Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
Bangkok, Krung Thep, Thailand

Background: Pediatric thromboembolism (TE) is a rare disease. In 2017, we established an International Pediatric Thrombosis Network (IPTN) to improve the care of children with TE. The IPTN currently consists of 92 pediatric thrombosis centers in 27 countries. Through the IPTN, we established a prospective disease registry, the Throm-PED registry, that brings together real world data on arterial (ATE) and venous TE (VTE) in children of all age groups globally.

Aims: To report the epidemiology, risk factors and management of children enrolled in the Throm-PED registry.

Methods: An international, multicentre, prospective observational cohort study of children ( < 18 years) with TE. The following data are prospectively collected in the basic registry: age at diagnosis, gender, TE type, location, risk factors, and treatment. In addition, the Throm-PED registry contains four sub-registers: safety and efficacy of DOACs, neonatal renal vein thrombosis, catheter-related ATE, and adolescent TE.

Results: The number of included children increased from 148 in 2019 (5 centers) to 895 in January 2022 (15 centers). Of 895 children, 795 had VTE (88.8%) and 100 had ATE (11.2%) (Table 1). Age distribution of VTE was bimodal with peaks in neonates and infants < 1 year and teenagers. Almost 80% of ATE children were younger than 8 years. Catheters, surgery, and congenital heart disease were more frequent in younger children with VTE, whereas thrombophilia, obesity, malignancy and immobility were more common in teenagers. Unprovoked VTE and infection were more common in males than females, whereas obesity and oral contraceptives were more frequent in females. DOACs were increasingly used, especially in teenagers.

Conclusion(s): The Throm-PED registry is an important tool to collect real world prospective data of pediatric TE which can provide post marketing surveillance data to validate previous clinical trials, identify geographic variations in clinical care, and assist in identifying optimal questions for future clinical trials.