Defibrotide for the treatment of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation Journal Article
| Authors: | Corbacioglu, S.; Kernan, N.; Lehmann, L.; Brochstein, J.; Revta, C.; Grupp, S.; Martin, P.; Richardson, P. G. |
| Article Title: | Defibrotide for the treatment of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation |
| 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. © 2012 Expert Reviews Ltd. |
| Keywords: | child; survival rate; busulfan; fludarabine; pathogenesis; review; drug dose comparison; drug withdrawal; hepatitis c; liver cirrhosis; liver transplantation; nonhuman; unspecified side effect; liver toxicity; bleeding; clinical assessment; palliative therapy; cyclophosphamide; melphalan; hematopoietic stem cell transplantation; carmustine; hypotension; liver; add on therapy; neuroblastoma; cardiovascular risk; pentoxifylline; heparin; anticoagulation; age distribution; immunosuppressive treatment; analgesia; hemodialysis; prostaglandin e1; glutamine; fibrinolytic agent; fibrinolytic agents; thymocyte antibody; multiple organ failure; gemtuzumab ozogamicin; alteplase; anticoagulant agent; danaparoid; bleeding tendency; endothelium injury; veno-occlusive disease; hepatic veno-occlusive disease; defibrotide; polydeoxyribonucleotides; ursodeoxycholic acid; fibrinolysis; sinusoidal obstruction syndrome; liver fibrosis; paracentesis; acetylcysteine; liver vein obstruction; platelet aggregation inhibitors; antithrombin iii; diuresis; albers schoenberg disease; lipoprostaglandin e1; griscelli syndrome; ventilator |
| Journal Title: | Expert Review of Hematology |
| Volume: | 5 |
| Issue: | 3 |
| ISSN: | 1747-4086 |
| Publisher: | Taylor & Francis Group |
| Date Published: | 2012-06-01 |
| Start Page: | 291 |
| End Page: | 302 |
| Language: | English |
| DOI: | 10.1586/ehm.12.18 |
| PROVIDER: | scopus |
| PUBMED: | 22780209 |
| DOI/URL: | |
| Notes: | --- - "Export Date: 1 August 2012" - "Source: Scopus" |
