PB0632 - Use of rADAMTS 13 in the treatment of TTP in children

Monday, June 24, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

Pawel Laguna, PhD MD

Professor of Medicine
Warsaw Medical Univesity, Department of Paediatric Oncology, Heamatology and Transplantology
Warsaw, Mazowieckie, Poland

Co-Author(s)

MS

Maria Szczepanska

Professor
Department of Pediatrics, Faculty of Medical Sciences in Zabrze, Medical University of Silesia in Katowice,
Zabrze, Slaskie, Poland
ET

Elzbieta Trembecka-Dubel

doctor
Department of Pediatrics, Faculty of Medical Sciences in Zabrze, Medical University of Silesia in Katowice,
Zabrze, Slaskie, Poland
JK

Joanna Kulik

doctor
Warsaw Medical Univesity, Department of Paediatric Oncology, Heamatology and Transplantology
Warsaw, Mazowieckie, Poland
MW

Magdalena Wojdalska

MD, PhD
Department of Pediatrics, Oncology and Hematology, Medical University of Lodz, Poland
Łódź, Lodzkie, Poland
AS

Andriy Sopilmyak

doctor
Department of Pediatrics, Oncology & Hematology Medical University of Lodz
lodz, Lodzkie, Poland
HB

Halina Bobrowska

doctor
SZOZ for Mather and Child Poznan
Poznan, Wielkopolskie, Poland
DP

Danuta Pietrys

doctor
University Children’s Hospital of Cracow
Krakow, Malopolskie, Poland
WB

Walentyna Balwierz

Professor
University Children’s Hospital of Cracow
krakow, Malopolskie, Poland

Background: TTP is a thrombotic microangiopathy, also described as an umbrella term for several disorders characterized clinically by the presence of microangiopathic haemolytic anaemia and thrombocytopenia, with pathological features including occlusive microvascular/macrovascular disease. TTP is caused by a severe deficiency of the plasma protease, as well as A disintegrin and Metalloprotease with a ThromboSpondin type 1 motif, member 13 (ADAMTS13).

Aims: Plasma is an effective treatment for exacerbations and for prophylaxis of TTP. However, we can now replace plasma with rADAMTS 13 as a treatment method.

Methods: The study included nine patients diagnosed with hereditary TTP. They were all between 0.6-10 years of age. Before entering the study, all patients were treated for exacerbations and prophylaxis with FFP. Allergic reactions occurred in 6 patients while using FFP
All patients were administered rADAMST 13 intravenously (IV) every 2 weeks at a dose of 40 IU per 1kg of body weight.

Results: During treatment with rADAMTS 13,observation period median 9 months, no adverse signs and symptoms were observed, and no disturbances were detected in morphological parameters of the blood, LDH or renal parameters. No thrombotic complications occurred in any patient.

Conclusion(s): TTP is still a disease that, if not diagnosed early, can be fatal in children.
The only treatment method used to be a prophylactic administration of FFP. The use of rADAMTS 13 is a safe and effective way to treat children with TTP.