Defibrotide for Patients with Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS): Interim Results From a Treatment IND Study

Highlights • Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) can be severe. • Severe VOD/SOS may be associated with multi-organ dysfunction and ≥80% mortality. • Defibrotide has demonstrated efficacy in treatment of severe hepatic VOD/SOS. • This has been the largest prospective eva...

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Published in:Biology of blood and marrow transplantation 2017-06, Vol.23 (6), p.997-1004
Main Authors: Richardson, Paul G., MD, Smith, Angela R., MD, MS, Triplett, Brandon M., MD, Kernan, Nancy A., MD, Grupp, Stephan A., MD, PhD, Antin, Joseph H., MD, Lehmann, Leslie, MD, Shore, Tsiporah, MD, Iacobelli, Massimo, MD, Miloslavsky, Maja, PhD, Hume, Robin, MS, Hannah, Alison L., MD, Nejadnik, Bijan, MD, Soiffer, Robert J., MD
Format: Article
Language:English
Subjects:
Defibrotide
Hematology, Oncology and Palliative Medicine
Hematopoietic stem cell transplantation
Multiorgan dysfunction
Sinusoidal obstruction syndrome
Veno-occlusive disease
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Summary:Highlights • Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) can be severe. • Severe VOD/SOS may be associated with multi-organ dysfunction and ≥80% mortality. • Defibrotide has demonstrated efficacy in treatment of severe hepatic VOD/SOS. • This has been the largest prospective evaluation of defibrotide for severe VOD/SOS.
ISSN:1083-8791
1523-6536
DOI:10.1016/j.bbmt.2017.03.008