Defibrotide
| Clinical data | |
|---|---|
| AHFS/Drugs.com | International Drug Names |
| Pregnancy category |
|
| Routes of administration |
Intravenous |
| ATC code | |
| Legal status | |
| Legal status |
|
| Pharmacokinetic data | |
| Bioavailability | 58 - 70% by mouth (i.v. and i.m. = 100%) |
| Biological half-life | t1/2-alpha = minutes; t1/2-beta = a few hours |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| ChemSpider |
|
| UNII | |
| KEGG | |
  (what is this?)
|
|
Defibrotide, sold under the brandname Defitelio, is a mixture of single-stranded oligonucleotides that is purified from the intestinal mucosa of pigs. It is used to treat veno-occlusive disease of the liver of people having had a bone marrow transplant. It works by protecting the cells lining bloods vessels and preventing blood clotting; the way it does this is not well understood.
There is a strong risk of bleeding in the brain, eyes, lungs, gastrointestinal tract, urinary tract, and nose. Some people have hypersensitivity reactions.
It was approved in Europe in 2013 and in the US in 2014.
Defibrotide is used to treat veno-occlusive disease of the liver of people having had a bone marrow transplant. As of 2016, however, randomized placebo controlled trials have not been done.
It is administered by intravenous infusion in a doctor's office or clinic.
Pregnant women should not take defibrotide and women should not become pregnant while taking it; it has not been tested in pregnant but at normal doses it caused hemolytic abortion in rats.
Use of defibrotide for people who are already taking anticoagulants is dangerous and use of other drugs that affect platelet aggregation, like NSAIDs, should be done with care. Defibrotide should not be given to people who have a difficult time maintaining a steady blood pressure.
There is a strong risk of hemorrhage and some people have had hypersensitivity reactions to defibrotide.
Common adverse effects, occurring in between 1 and 10% of people, included impaired blood clotting, cerebral hemorrhage, bleeding in the eyes, low blood pressure, bleeding lungs, bleeding stomach or intestines, vomiting, blood in the urine, and bleeding at catheterization sites.
Other side effects have included diarrhea, nosebleeds, sepsis, graft vs host disease, pneumonia, and infections.
...
Wikipedia