defibrotide at a glance
Medicine class
Antithrombotic agent with endothelial-protective and profibrinolytic effects
Licensed use
Severe hepatic veno-occlusive disease after stem-cell transplantation
Who can receive it
Adults and children, including infants older than one month
Administration
Diluted and given into a vein under specialist hospital supervision
Key safety concern
Bleeding, especially with medicines that affect clotting
What is defibrotide used for?
Defibrotide is licensed for:
Treating severe hepatic veno-occlusive disease, also known as sinusoidal obstruction syndrome, associated with haematopoietic stem-cell transplantation in adults and children older than one month.
It should not be used routinely to prevent veno-occlusive disease after stem-cell transplantation.
How does defibrotide work?
Defibrotide is a mixture of oligonucleotides. It reduces excessive activation and injury of blood-vessel linings, limits platelet and white-cell adhesion, and supports fibrin breakdown. Its exact mechanism is not fully established.
defibrotide preparations and strengths
Solution for infusion
Route: Intravenous
Strengths: 80 mg/mL
How to take defibrotide
Defibrotide is prescribed and administered by clinicians experienced in stem-cell transplant complications. The concentrate is diluted before intravenous infusion. Vials are single-use and unused contents are discarded.
Tell the team about bleeding, planned surgery or invasive procedures.
Ensure staff know about every anticoagulant, antiplatelet, thrombolytic and anti-inflammatory medicine being used.
defibrotide side effects
Common or expected effects
- Nausea, vomiting or diarrhoea
- Rash or itching
- Fever
- Nosebleed, mouth bleeding, skin spots or bleeding around the infusion catheter
Get urgent medical advice
- Major internal or external bleeding, including gastrointestinal, lung or brain bleeding
- Low blood pressure causing faintness or circulatory instability
- Severe allergic reaction with swelling or breathing difficulty
- Disturbance of blood clotting
defibrotide in pregnancy
Human pregnancy data are unavailable. Defibrotide should generally not be used during pregnancy unless the woman's clinical condition requires it. Effective contraception is required for patients and their partners during exposure and for one week afterwards.
defibrotide while breastfeeding
It is unknown whether defibrotide passes into human milk. The product information considers risk to the infant unlikely and permits use during breastfeeding, but the specialist team should review individual circumstances.
defibrotide interactions
Interactions mainly involve an increased bleeding risk.
Thrombolytics, such as tissue plasminogen activator
Concurrent use is contraindicated because combined profibrinolytic effects may increase haemorrhage.
Systemic anticoagulants, including heparins, warfarin, dabigatran, rivaroxaban and apixaban
Combined use is not recommended. Exceptional use requires close monitoring.
Antiplatelet medicines and NSAIDs, including aspirin, ibuprofen, naproxen and diclofenac
These may increase bleeding risk and require caution and close medical supervision.
Common questions about defibrotide
Answers are fully visible for fast scanning and source review.
What is defibrotide used for?
It treats severe hepatic veno-occlusive disease, also called sinusoidal obstruction syndrome, following haematopoietic stem-cell transplantation.
Is it used to prevent veno-occlusive disease?
Routine preventive use after stem-cell transplantation is not recommended; the licence is for established severe disease.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.