Defibrotide: A Review of Its Use in Severe Hepatic Veno-Occlusive Disease Following Haematopoietic Stem Cell Transplantation

  title={Defibrotide: A Review of Its Use in Severe Hepatic Veno-Occlusive Disease Following Haematopoietic Stem Cell Transplantation},
  author={Gillian M. Keating},
  journal={Clinical Drug Investigation},
  • G. Keating
  • Published 29 October 2014
  • Medicine, Biology
  • Clinical Drug Investigation
Defibrotide (Defitelio®) was recently approved in the EU for the treatment of severe hepatic veno-occlusive disease (VOD), also known as sinusoidal obstructive syndrome, in haematopoietic stem cell transplantation (HSCT) therapy. It is indicated in adults, adolescents, children and infants over 1 month of age. Defibrotide is also available in the US via an expanded-access protocol. Defibrotide is thought to protect endothelial cells and restore the thrombo-fibrinolytic balance in VOD. In a… 
Defibrotide for the treatment and prophylaxis of hepatic veno-occlusive disease
The current evidence is not sufficient to evaluate the effectiveness and safety of defibrotide for the treatment of VOD, and an inclusion in the Austrian catalogue of benefits for an off-label indication cannot be recommended.
Defibrotide in the treatment of hepatic veno-occlusive disease
Defibrotide (DF) has been proposed for the treatment of SOS due to its ability to restore thrombo-fibrinolytic balance and protect endothelial cells.
Defibrotide: Real World Management of Veno-Occlusive Disease/ Sinusoidal Obstructive Syndrome after Stem Cell Transplant.
Early diagnosis, prompt initiation of defibrotide, and minimizing dosing interruptions may be key to successful treatment of VOD/ SOS.
Identification and Management: Sinusoidal obstruction syndrome/veno‐occlusive disease related to hematopoietic stem cell transplantation
This article describes the syndrome, risk factors, signs and symptoms, and appropriate supportive care and treatment of Sinusoidal obstruction syndrome and offers some practical tips for detecting SOS/VOD and providing patient care.
The budget impact and cost-effectiveness of defibrotide for treatment of veno-occlusive disease with multi-organ dysfunction in patients post-hematopoietic stem cell transplant
The budget impact of defibrotide for a transplant center is relatively modest compared to the overall cost of transplantation, and the life years gained lead to defib rotide being highly cost-effective.
Outcomes of combination treatment with MARS and TIPS for hepatic veno-occlusive disease: a report of 12 cases
The combination of MARS and TIPS creation is promising as a potential treatment for acute HVOD, and it showed an improvement in overall survival.
Defibrotide enhances fibrinolysis in human endotoxemia – a randomized, double blind, crossover trial in healthy volunteers
In conclusion, defibrotide infusion enhanced fibrinolysis and reduced C-reactive protein levels during experimental endotoxemia and suggests modest anticoagulant properties.
Early and Acute Complications and the Principles of HSCT Nursing Care
  • E. WallhultB. Quinn
  • Medicine
    The European Blood and Marrow Transplantation Textbook for Nurses
  • 2017
Recommendations for prevention and principles for nursing care are presented since careful nursing monitoring, prompt intervention and care may have an influence on patients’ morbidity and mortality.


Safety and efficacy of defibrotide for the treatment of severe hepatic veno-occlusive disease
Defibrotide, a polydisperse oligonucleotide, has been shown in phase II and III trials to improve complete response and survival in patients undergoing SCT with severe VOD.
Defibrotide in the treatment of children with veno-occlusive disease (VOD): a retrospective multicentre study demonstrates therapeutic efficacy upon early intervention
Defibrotide (DF) is a polydeoxyribonucleotide with aptameric activity on endothelium that is effective in treatingeno-occlusive disease of the liver and in multivariate analysis, early intervention remained the only significant factor for a CR.
Hepatic veno-occlusive disease after hematopoietic stem cell transplantation: review and update on the use of defibrotide.
A pivotal, prospective, multinational, phase III trial of DF is underway in patients with severe VOD, and should provide validation of this agent as a therapy for established disease with a high risk of mortality.
Defibrotide for the treatment of severe hepatic veno-occlusive disease and multiorgan failure after stem cell transplantation: a multicenter, randomized, dose-finding trial.
  • P. RichardsonR. Soiffer E. Guinan
  • Medicine, Biology
    Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
  • 2010
Safety of Defibrotide (DF) In Stem Cell Transplant (SCT) Patients (Pts).
A comprehensive review of safety for DF in this setting, including a review of the contribution of DF to observed toxicities in the initial single-arm trials, found that DF was generally well tolerated.
Prophylactic defibrotide in allogeneic stem cell transplantation: minimal morbidity and zero mortality from veno-occlusive disease
It is suggested that prophylaxis with defibrotide alone may reduce the incidence of VOD post-SCT although a randomised controlled trial is warranted to further evaluate its role.
Safety and effects of prophylactic defibrotide for sinusoidal obstruction syndrome in hematopoietic stem cell transplantation.
It could be effective to use prophylactic defibrotide in advance to improve HSCT outcomes in patients at risk of SOS, according to this study.
Multi-institutional use of defibrotide in 88 patients after stem cell transplantation with severe veno-occlusive disease and multisystem organ failure: response without significant toxicity in a high-risk population and factors predictive of outcome.
The complete response rate, survival to day +100, and absence of significant DF-associated toxicity in this largest patient cohort reported to date confirm the results of earlier studies.