Corbacioglu, Selim and Richardson, Paul G. (2017) Defibrotide for children and adults with hepatic veno-occlusive disease post hematopoietic cell transplantation. EXPERT REVIEW OF GASTROENTEROLOGY & HEPATOLOGY, 11 (10). pp. 885-898. ISSN 1747-4124, 1747-4132
Full text not available from this repository. (Request a copy)Abstract
Introduction: Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a complication that is typically associated with conditioning for hematopoietic stem cell transplantation (HSCT). In patients with concomitant multi-organ dysfunction, mortality may be >80%. Recently, the European Society for Blood and Marrow Transplantation established separate criteria for diagnosis and severity of VOD/SOS for adults and children, to better reflect current understanding of the disease. Areas covered: This review provides an overview of post-HSCT hepatic VOD/SOS and defibrotide, including its pharmacological, clinical, and regulatory profile. In children and adults following HSCT, defibrotide is approved for the treatment of hepatic VOD/SOS with concomitant renal or pulmonary dysfunction in the United States and for the treatment of severe hepatic VOD/SOS in the European Union. Day +100 survival rates with defibrotide are superior to those of historical controls receiving best supportive care only, and safety profiles are similar. Expert commentary: Defibrotide appears to act through multiple mechanisms to restore thrombofibrinolytic balance and protect endothelial cells, and there are promising data on the use of defibrotide for VOD/SOS prophylaxis in high-risk children undergoing HSCT. An ongoing randomized controlled trial in children and adults will better assess the clinical value of defibrotide as a preventive medication.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | BONE-MARROW-TRANSPLANTATION; SYNDROME/VENO-OCCLUSIVE DISEASE; HIGH-RISK POPULATION; ENDOTHELIAL-CELLS; OBSTRUCTION SYNDROME; COMPASSIONATE-USE; URSODEOXYCHOLIC ACID; SIGNIFICANT TOXICITY; MULTIORGAN FAILURE; EUROPEAN-SOCIETY; Defibrotide; endothelial cell activation; fibrinolysis; hematopoietic stem cell transplantation; hepatic veno-occlusive disease; multi-organ dysfunction; multi-organ failure; sinusoidal obstruction syndrome; thrombosis |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 14 Dec 2018 13:01 |
| Last Modified: | 20 Feb 2019 14:25 |
| URI: | https://pred.uni-regensburg.de/id/eprint/614 |
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