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.

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

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去纤维肽治疗未接受造血干细胞移植的儿童静脉闭塞性疾病/鼻窦阻塞综合征：来自德国一家学术医院的病例报告

静脉闭塞性疾病/窦状静脉阻塞综合征（VOD/SOS）是一种潜在的危及生命的疾病，其特征是肝脏小静脉阻塞。虽然通常与造血干细胞移植相关，但VOD/SOS也可能在强化多模式化疗方案后发生。在儿童中，VOD/SOS的症状是难治性血小板减少，体重增加，肝肿大，腹水和液体潴留，高胆红素血症，有时右上腹疼痛。方法：在此，我们报告了6例急性淋巴细胞白血病患儿在接受标准aiop - bfm ALL方案治疗时发生严重VOD/SOS的病例系列。结果/结论：所有患者对去纤维肽治疗反应迅速，并表现出良好的临床结果。试验注册：作者已确认本次提交不需要临床试验注册。

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EJHaem

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