A medicine for severe hepatic veno-occlusive disease after a transplant
Defibrotide
A specialist hospital medicine used to treat severe hepatic veno-occlusive disease (a serious liver complication) after a stem-cell transplant.
What is Defibrotide?
Defibrotide is a specialist medicine given in hospital to treat severe hepatic veno-occlusive disease, also called sinusoidal obstruction syndrome, a serious complication in which small blood vessels in the liver become blocked after a stem-cell (bone-marrow) transplant. It is given into a vein by the transplant team. The most important risk is bleeding (haemorrhage), so a crucial safety point is that it must not be used together with other clot-busting or blood-thinning medicines, and the team watches closely for any bleeding. Serious allergic reactions can also occur.
Education and reference only. This is a plain-language guide to Defibrotide — it deliberately contains no doses. Doses depend on the person, the brand and the reason for treatment, and belong with your prescriber. Always check the BNF, the product labelling (SmPC) and follow medical advice.
What it is
Defibrotide is a specialist medicine used to treat severe hepatic veno-occlusive disease, also known as sinusoidal obstruction syndrome. This is a serious complication that can follow a stem-cell (bone-marrow) transplant, in which the tiny blood vessels inside the liver become damaged and blocked, causing the liver to swell, fluid to build up and the liver to work poorly. Defibrotide is given as a drip into a vein over several days by the transplant team in hospital. It is a treatment for an established, serious problem rather than something used at home.
How it works
In hepatic veno-occlusive disease, the lining of the small blood vessels in the liver is injured and clots and blockages form, so blood cannot flow through the liver properly. Defibrotide is thought to protect and help repair this vessel lining and to help keep the small vessels open, improving blood flow through the liver while keeping a careful balance so as not to cause too much bleeding. Because it acts on the blood-vessel lining and the clotting system, it carries a risk of bleeding, which is why it is given and monitored carefully in hospital.
Company & origin
Originated / developed by: Specialist manufacturers.
A specialist hospital medicine used in the UK to treat severe hepatic veno-occlusive disease after a stem-cell transplant.
Practical use
How to take Defibrotide
General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.
- It is given as a drip into a vein in hospital by the transplant team, so it is fully managed for you.
- Tell the team about every medicine you take, especially any blood thinners or clot-busting medicines, as these must not be used at the same time.
- Report any signs of bleeding straight away, such as unusual bruising, blood in the urine or stools, or bleeding that will not stop.
- Let the team know if you feel unwell during the drip, such as flushing, breathlessness, rash or swelling, as these can signal a reaction.
- Tell the team about any planned operations or procedures, as the timing of treatment may need to be adjusted.
Weighing it up
Advantages & disadvantages of Defibrotide
Advantages
- Treats severe hepatic veno-occlusive disease, a serious liver complication after a stem-cell transplant.
- Works on the damaged blood vessels in the liver to help restore blood flow.
- Given and closely monitored by an expert transplant team in hospital.
Disadvantages
- Carries a risk of bleeding (haemorrhage), which needs close monitoring.
- Must not be used together with other blood-thinning or clot-busting medicines.
- Can cause allergic-type reactions, including serious ones, during the drip.
Practical use
Good to know
Defibrotide is a serious-illness treatment given only in hospital by an expert transplant team, so it is fully managed for you. The most important risk to understand is bleeding (haemorrhage): because the medicine affects the clotting system, the team watches closely for any signs of bleeding, and a crucial rule is that it must not be given at the same time as other medicines that thin the blood or break down clots, as together they greatly increase the bleeding risk. The team will pause or stop these other medicines and may delay defibrotide around surgery or procedures. Allergic-type reactions, including serious ones, can also happen during the drip, so you are watched while it is given. Tell the team about all your medicines and any bleeding problems.
Who should not take it / use with caution
- It must not be used at the same time as other medicines that thin the blood or break down clots, because of the high bleeding risk.
- It should not be used in people with significant active bleeding.
- People who have had a serious allergic reaction to defibrotide should not be given it.
Monitoring
- Close watching for any signs of bleeding throughout treatment.
- Checking blood pressure and signs of allergic reactions during the drip.
- Reviewing liver function and how the veno-occlusive disease is responding.
Side effects
- Bleeding, which can range from minor bruising to more serious haemorrhage and is watched for closely.
- A drop in blood pressure.
- Feeling sick, being sick or diarrhoea.
- Allergic-type reactions, including, rarely, serious ones during the drip, which need urgent attention.
Key interactions
- Medicines that thin the blood, such as heparins or other anticoagulants, must not be used at the same time.
- Clot-busting (thrombolytic) medicines greatly increase the bleeding risk and must be avoided together.
- Other medicines that affect clotting or platelets are reviewed carefully by the transplant team.
Available as: A concentrate made up into a drip given into a vein in hospital.
Answers
Defibrotide: frequently asked questions
What is defibrotide used for?
It is a hospital medicine used to treat severe hepatic veno-occlusive disease (sinusoidal obstruction syndrome), a serious liver complication after a stem-cell transplant.
What is the main risk?
The main risk is bleeding (haemorrhage), because the medicine affects the clotting system, so the team watches closely for any signs of bleeding.
Can it be used with blood thinners?
No. It must not be used at the same time as blood-thinning or clot-busting medicines, as together they greatly increase the risk of bleeding.
How is it given?
It is given as a drip into a vein in hospital over several days by the transplant team, so it is fully managed for you.
What should I watch for during treatment?
Tell the team straight away about any unusual bruising or bleeding, or if you feel flushed, breathless or develop a rash during the drip.
Authoritative sources
- BNF
- NICE CKS
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