Corbacioglu, Selim and Kernan, Nancy and Lehmann, Leslie and Brochstein, Joel and Revta, Carolyn and Grupp, Stephan and Martin, Paul and Richardson, Paul G. (2012) Defibrotide for the treatment of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation. EXPERT REVIEW OF HEMATOLOGY, 5 (3). pp. 291-302. ISSN 1747-4086
Full text not available from this repository. (Request a copy)Abstract
Hepatic veno-occlusive disease (VOD) is a serious complication of stem cell transplantation in children. VOD is characterized by rapid weight gain, hepatomegaly, hyperbilirubinemia and ascites. The pathogenesis of VOD is thought to involve chemotherapy and radiation-induced damage to the sinusoidal endothelium, resulting in endothelial injury, microthrombosis, subendothelial damage and cytokine activation. These processes lead to concomitant progressive hepatocellular dysfunction and subsequent fluid retention and renal impairment. Severe VOD is typically associated with multiorgan failure and high mortality. A number of possible strategies for the prevention and/or treatment of VOD in children have been investigated. The most promising agent to date is defibrotide, a novel polydeoxyribonucleotide with fibrinolytic properties but no major bleeding risk. Numerous studies, including Phase II/III trials, have shown clinical benefit in pediatric patients with the use of defibrotide treatment and prophylaxis. This review discusses VOD in children and focuses on therapeutic options, including defibrotide, in this patient population.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | BONE-MARROW-TRANSPLANTATION; SINUSOIDAL OBSTRUCTION SYNDROME; TISSUE-PLASMINOGEN ACTIVATOR; REGIMEN-RELATED TOXICITY; HIGH-RISK POPULATION; IN-VITRO; GEMTUZUMAB OZOGAMICIN; URSODEOXYCHOLIC ACID; INTRAVENOUS BUSULFAN; VENOCCLUSIVE DISEASE; defibrotide; sinusoidal obstruction syndrome; veno-occlusive disease |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Petra Gürster |
| Date Deposited: | 15 May 2020 10:17 |
| Last Modified: | 15 May 2020 10:17 |
| URI: | https://pred.uni-regensburg.de/id/eprint/18614 |
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