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Defibrotide sodium for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome.

Publication ,  Journal Article
Richardson, PG; Triplett, BM; Ho, VT; Chao, N; Dignan, FL; Maglio, M; Mohty, M
Published in: Expert Rev Clin Pharmacol
February 2018

Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is an unpredictable condition associated with endothelial-cell damage due to conditioning for hematopoietic stem-cell transplantation (HSCT) or chemotherapy without HSCT. Mortality in patients with VOD/SOS and multi-organ dysfunction (MOD) may be >80%. Areas covered: Defibrotide is the only approved drug for the treatment of severe hepatic VOD/SOS after HSCT in the European Union and hepatic VOD/SOS with renal or pulmonary dysfunction in the United States. Its efficacy in patients with VOD/SOS with MOD post-HSCT was demonstrated in a clinical-trial program that included a historically controlled treatment study, a phase 2 trial, and a large T-IND expanded-access program that also included patients without MOD and who received chemotherapy without HSCT. Expert commentary: Defibrotide appears to protect endothelial cells and restore the thrombolytic-fibrinolytic balance. It addresses a significant clinical need and has demonstrated favorable Day +100 survival and overall adverse-event rates that seem similar to control groups receiving supportive care alone. Currently, defibrotide is under investigation for the prevention of VOD/SOS in high-risk pediatric and adult patients.

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Published In

Expert Rev Clin Pharmacol

DOI

EISSN

1751-2441

Publication Date

February 2018

Volume

11

Issue

2

Start / End Page

113 / 124

Location

England

Related Subject Headings

  • Severity of Illness Index
  • Polydeoxyribonucleotides
  • Pharmacology & Pharmacy
  • Humans
  • Hepatic Veno-Occlusive Disease
  • Hematopoietic Stem Cell Transplantation
  • Fibrinolytic Agents
  • Endothelial Cells
  • Antineoplastic Agents
  • 3214 Pharmacology and pharmaceutical sciences
 

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Richardson, P. G., Triplett, B. M., Ho, V. T., Chao, N., Dignan, F. L., Maglio, M., & Mohty, M. (2018). Defibrotide sodium for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome. Expert Rev Clin Pharmacol, 11(2), 113–124. https://doi.org/10.1080/17512433.2018.1421943
Richardson, Paul G., Brandon M. Triplett, Vincent T. Ho, Nelson Chao, Fiona L. Dignan, Michelle Maglio, and Mohamad Mohty. “Defibrotide sodium for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome.Expert Rev Clin Pharmacol 11, no. 2 (February 2018): 113–24. https://doi.org/10.1080/17512433.2018.1421943.
Richardson PG, Triplett BM, Ho VT, Chao N, Dignan FL, Maglio M, et al. Defibrotide sodium for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome. Expert Rev Clin Pharmacol. 2018 Feb;11(2):113–24.
Richardson, Paul G., et al. “Defibrotide sodium for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome.Expert Rev Clin Pharmacol, vol. 11, no. 2, Feb. 2018, pp. 113–24. Pubmed, doi:10.1080/17512433.2018.1421943.
Richardson PG, Triplett BM, Ho VT, Chao N, Dignan FL, Maglio M, Mohty M. Defibrotide sodium for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome. Expert Rev Clin Pharmacol. 2018 Feb;11(2):113–124.
Journal cover image

Published In

Expert Rev Clin Pharmacol

DOI

EISSN

1751-2441

Publication Date

February 2018

Volume

11

Issue

2

Start / End Page

113 / 124

Location

England

Related Subject Headings

  • Severity of Illness Index
  • Polydeoxyribonucleotides
  • Pharmacology & Pharmacy
  • Humans
  • Hepatic Veno-Occlusive Disease
  • Hematopoietic Stem Cell Transplantation
  • Fibrinolytic Agents
  • Endothelial Cells
  • Antineoplastic Agents
  • 3214 Pharmacology and pharmaceutical sciences