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Kleinschmidt, Katharina ; Troeger, Anja ; Föll, Jürgen ; Hanafee‐Alali, Tarek ; Jakob, Marcus ; Kramer, Sonja ; Kietz, Silke ; Corbacioglu, Selim

Defibrotide for the Treatment of Veno‐Occlusive Disease/Sinusoidal Obstruction Syndrome in Paediatric Patients Who Did Not Receive Haematopoietic Stem Cell Transplantation: Case Reports of Patients From a German Academic Hospital

Article

Kleinschmidt, Katharina , Troeger, Anja, Föll, Jürgen, Hanafee‐Alali, Tarek, Jakob, Marcus, Kramer, Sonja, Kietz, Silke and Corbacioglu, Selim (2025) Defibrotide for the Treatment of Veno‐Occlusive Disease/Sinusoidal Obstruction Syndrome in Paediatric Patients Who Did Not Receive Haematopoietic Stem Cell Transplantation: Case Reports of Patients From a German Academic Hospital. eJHaem 6 (1), e1094.

DOI to cite this document: 10.5283/epub.74758


Abstract

Introduction: Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening condition characterised by obstruction of the small veins of the liver. Although typically associated with haematopoietic stem cell transplantation, VOD/SOS may also occur following intensive multimodal chemotherapy regimens. In children, symptoms of VOD/SOS are refractory ...

Introduction:
Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening condition characterised by obstruction of the small veins of the liver. Although typically associated with haematopoietic stem cell transplantation, VOD/SOS may also occur following intensive multimodal chemotherapy regimens. In children, symptoms of VOD/SOS are refractory thrombocytopaenia, weight gain, hepatomegaly, ascites and fluid retention, hyperbilirubinaemia and sometimes right upper quadrant pain.

Methods:
Here, we present a case series of six paediatric patients with acute lymphoblastic leukaemia who developed severe VOD/SOS while receiving standard AIEOP-BFM ALL protocol treatment.

Results/Conclusions:
All patients responded promptly to defibrotide treatment and exhibited favourable clinical outcomes.

Trial Registration:
The authors have confirmed clinical trial registration is not needed for this submission.



Involved Institutions


Details

Item typeArticle
Journal or Publication TitleeJHaem
PublisherWiley
Open Access TypeDEAL (Wiley Gold)
Volume6
Number of Issue or Book Chapter1
Page Rangee1094
Date23 January 2025
Date of publication27 Jan 2025 05:32
InstitutionsMedicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation
Identification Number
ValueType
10.1002/jha2.1094DOI
Keywordsacute lymphoblastic leukaemia | chemotherapy | children | defibrotide | pEBMT diagnostic criteria for VOD/SOS | veno-occlusive disease/sinusoidal obstruction syndrome
Dewey Decimal Classification600 Technology > 610 Medical sciences Medicine
StatusPublished
RefereedYes, this version has been refereed
Created at the University of RegensburgYes
URN of the UB Regensburgurn:nbn:de:bvb:355-epub-747588
Item ID74758

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