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Defibrotide for the treatment of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation.

Publication ,  Journal Article
Corbacioglu, S; Kernan, N; Lehmann, L; Brochstein, J; Revta, C; Grupp, S; Martin, P; Richardson, PG
Published in: Expert Rev Hematol
June 2012

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.

Duke Scholars

Published In

Expert Rev Hematol

DOI

EISSN

1747-4094

Publication Date

June 2012

Volume

5

Issue

3

Start / End Page

291 / 302

Location

England

Related Subject Headings

  • Polydeoxyribonucleotides
  • Platelet Aggregation Inhibitors
  • Liver
  • Humans
  • Hepatic Veno-Occlusive Disease
  • Hematopoietic Stem Cell Transplantation
  • Fibrinolytic Agents
  • Child
  • 3201 Cardiovascular medicine and haematology
  • 1199 Other Medical and Health Sciences
 

Citation

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Corbacioglu, S., Kernan, N., Lehmann, L., Brochstein, J., Revta, C., Grupp, S., … Richardson, P. G. (2012). Defibrotide for the treatment of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation. Expert Rev Hematol, 5(3), 291–302. https://doi.org/10.1586/ehm.12.18
Corbacioglu, Selim, Nancy Kernan, Leslie Lehmann, Joel Brochstein, Carolyn Revta, Stephan Grupp, Paul Martin, and Paul G. Richardson. “Defibrotide for the treatment of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation.Expert Rev Hematol 5, no. 3 (June 2012): 291–302. https://doi.org/10.1586/ehm.12.18.
Corbacioglu S, Kernan N, Lehmann L, Brochstein J, Revta C, Grupp S, et al. Defibrotide for the treatment of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation. Expert Rev Hematol. 2012 Jun;5(3):291–302.
Corbacioglu, Selim, et al. “Defibrotide for the treatment of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation.Expert Rev Hematol, vol. 5, no. 3, June 2012, pp. 291–302. Pubmed, doi:10.1586/ehm.12.18.
Corbacioglu S, Kernan N, Lehmann L, Brochstein J, Revta C, Grupp S, Martin P, Richardson PG. Defibrotide for the treatment of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation. Expert Rev Hematol. 2012 Jun;5(3):291–302.

Published In

Expert Rev Hematol

DOI

EISSN

1747-4094

Publication Date

June 2012

Volume

5

Issue

3

Start / End Page

291 / 302

Location

England

Related Subject Headings

  • Polydeoxyribonucleotides
  • Platelet Aggregation Inhibitors
  • Liver
  • Humans
  • Hepatic Veno-Occlusive Disease
  • Hematopoietic Stem Cell Transplantation
  • Fibrinolytic Agents
  • Child
  • 3201 Cardiovascular medicine and haematology
  • 1199 Other Medical and Health Sciences