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 Pub Date : 2025-01-23 DOI:10.1002/jha2.1094

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

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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 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.