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Defibrotide: An Oligonucleotide for Sinusoidal Obstruction Syndrome
May T Aziz1,2, Payal P Kakadiya1, Samantha M Kush1
11 Virginia Commonwealth University Health System/Medical College of Virginia Hospitals, Richmond, VA, USA.
The Annals of Pharmacotherapy
|September 16, 2017
Summary
Defibrotide is effective and safe for treating hepatic veno-occlusive disease (VOD/SOS) after hematopoietic cell transplantation (HCT). Its prophylactic use is debated due to cost-effectiveness concerns, despite some guideline recommendations.
Area of Science:
- Pharmacology and Therapeutics
- Hematology
- Oncology
Background:
- Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), is a serious complication following hematopoietic cell transplantation (HCT).
- Defibrotide is a novel therapeutic agent investigated for VOD/SOS management.
Purpose of the Study:
- To review the efficacy, safety, pharmacology, pharmacokinetics, and clinical utility of defibrotide for VOD/SOS.
- To evaluate the role of defibrotide in both treatment and prophylaxis post-HCT.
Main Methods:
- Comprehensive literature search of PubMed, clinicaltrials.gov, and other sources up to August 2017.
- Inclusion of English-language trials assessing defibrotide's pharmacodynamics, efficacy, safety, and cost-effectiveness.
Main Results:
- Defibrotide demonstrated significant efficacy and safety in treating VOD/SOS in adult and pediatric HCT patients, with notable response and survival rates.
- Guideline recommendations support defibrotide prophylaxis in high-risk HCT patients, but its cost-effectiveness remains controversial.
- Defibrotide exhibits manageable toxicities with a low incidence of severe adverse events.
Conclusions:
- Defibrotide is the first FDA-approved treatment for hepatic VOD/SOS with organ dysfunction post-HCT.
- Conflicting data and lack of proven cost-effectiveness limit the current understanding of defibrotide's prophylactic role.
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