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. 2025 Jan 23;6(1):e1094.
doi: 10.1002/jha2.1094. eCollection 2025 Feb.

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

Katharina Kleinschmidt  1 , Anja Troeger  1 , Jürgen Föll  1 , Tarek Hanafee-Alali  1 , Marcus Jakob  1 , Sonja Kramer  1 , Silke Kietz  1 , Selim Corbacioglu  1
Affiliations

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

Katharina Kleinschmidt et al. EJHaem. .

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.

Keywords: acute lymphoblastic leukaemia; chemotherapy; children; defibrotide; pEBMT diagnostic criteria for VOD/SOS; veno‐occlusive disease/sinusoidal obstruction syndrome.

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Conflict of interest statement

Katharina Kleinschmidt received support for participation in an advisory board from Jazz Pharmaceuticals and congress sponsorship from Medac GmbH. Selim Corbacioglu received honorarium for advisory activity from Jazz Pharmaceuticals. The other authors declare no conflicts of interest.

References

    1. Dalle J. H. and Giralt S. A., “Hepatic Veno‐Occlusive Disease After Hematopoietic Stem Cell Transplantation: Risk Factors and Stratification, Prophylaxis, and Treatment,” Biology of Blood and Marrow Transplantation 22, no. 3 (2016): 400–409. - PubMed
    1. Corbacioglu S., Carreras E., Ansari M., et al., “Diagnosis and Severity Criteria for Sinusoidal Obstruction Syndrome/Veno‐Occlusive Disease in Pediatric Patients: A New Classification From the European Society for Blood and Marrow Transplantation,” Bone Marrow Transplantation 53, no. 2 (2018): 138–145. - PMC - PubMed
    1. Cairo M. S., Cooke K. R., Lazarus H. M., and Chao N., “Modified Diagnostic Criteria, Grading Classification and Newly Elucidated Pathophysiology of Hepatic SOS/VOD After Haematopoietic Cell Transplantation,” British Journal of Haematology 190, no. 6 (2020): 822–836. - PMC - PubMed
    1. Kernan N. A., Richardson P. G., Smith A. R., et al., “Defibrotide for the Treatment of Hepatic Veno‐Occlusive Disease/Sinusoidal Obstruction Syndrome Following Nontransplant‐Associated Chemotherapy: Final Results From a Post Hoc Analysis of Data From an Expanded‐Access Program,” Pediatric Blood & Cancer 65, no. 10 (2018): e27269. - PMC - PubMed
    1. Wang X., Benettaib B., Ni W., et al., “Veno‐Occlusive Disease/Sinusoidal Obstruction Syndrome Without Hematopoietic Cell Transplantation in a Real‐World Population in the United States: Patient Characteristics, Prior Treatment Patterns, and Time to Diagnosis,” Blood 138, no. Supplement 1 (2021): 1946.

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