An oligonucleotide with a mechanism of action encompassing both restoration of the thrombo-fibrolytic balance and endothelial cell protection.
Approved in patients with hepatic VOD with multiple organ dysfunction following hematopoietic stem cell transplant.
Defibrotide is a mixture of single-stranded oligonucleotides that is purified from the intestinal mucosa of pigs.
Brand name Defitelio.
It is used to treat veno-occlusive disease following a bone marrow transplant.
It is used to treat veno-occlusive disease of the liver of people having had a bone marrow transplant.
It works by protecting the endothelium.
Pregnancy category X.
Routes of administration- IV
Bioavailability 58 – 70% by mouth.
Elimination half-life < 2 hours.
It is associated with risk of bleeding in the brain, eyes, lungs, gastrointestinal tract, urinary tract, and nose.
It is administered by intravenous infusion.
Pregnant women should not take defibrotide
Its use in people who are already taking anticoagulants or antiplatelet drug is dangerous.
Some patients have had hypersensitivity reactions.
Common adverse effects, occurring in between 1 and 10% of people, include: impaired blood clotting, vomiting, low blood pressure, bleeding in the nose, brain, eyes, lungs, stomach or intestines, in the urine, and at catheterization sites.