Defibrotide
Defibrotide is an antithrombotic medication primarily used to treat severe hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome, in patients who develop renal or pulmonary dysfunction following hematopoietic stem cell transplantation. It works by enhancing the enzymatic activity of plasmin to help break down fibrin clots in the small blood vessels of the liver.
Defibrotide treats severe hepatic veno-occlusive disease (VOD/SOS) by enhancing plasmin to break down fibrin clots in liver blood vessels.
Key facts at a glance
- ATC
- B01AX01
- RxNorm
- 1311089
- Generic
- Defibrotide
- Class
- atc-b01
- Brands
- Defitelio
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 6.25 mg/kg every 6 hours
- Maximum
- consult physician
- Frequency
- every 6 hours (25 mg/kg/day total)
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 6.25 mg/kg every 6 hours
- Maximum
- consult physician
- Frequency
- every 6 hours (25 mg/kg/day total)
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 6.25 mg/kg every 6 hours
- Maximum
- consult physician
- Frequency
- every 6 hours (25 mg/kg/day total)
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
8 groupspregnancy
first trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks
regular fetal monitoring
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
second third trimesteruse only if clearly needed
use only when benefits outweigh potential risks to fetus
regular fetal monitoring and ultrasound assessments
Use with caution and monitor for adverse effects.
lactation
breastfeedingdiscontinue breastfeeding
breastfeeding should be discontinued during treatment due to unknown excretion in breast milk
monitor infant for any adverse effects
pediatric_age
children adolescentsweight-based dosing
approved for use in pediatric patients with hepatic VOD with renal or pulmonary dysfunction
monitor for bleeding, renal function, and hepatic function
geriatric
elderly over 65no specific dose adjustment
use with caution due to limited clinical data in elderly population
monitor renal function and for signs of bleeding
Use with caution and monitor for adverse effects.
renal_impairment
mild moderateno specific adjustment recommended
use with caution, monitor renal function closely
regular monitoring of serum creatinine and renal function
Use with caution and monitor for adverse effects.
renal_impairment
severeconsider dose reduction
use with extreme caution, consider reduced dose in severe renal impairment
frequent monitoring of renal function and drug levels if available
Use with caution and monitor for adverse effects.
hepatic_impairment
moderate severeno specific adjustment for hepatic VOD
defibrotide is specifically indicated for treatment of hepatic VOD; monitor liver function tests
monitor liver function tests, bilirubin, and for signs of bleeding
Use with caution and monitor for adverse effects.
Statistics and clinical data
FDA approval year
FDA approval history for Defibrotide
Drug class
Pharmacological classification
Primary indication
Therapeutic indication
Route of administration
Dosage form and route
Treatment of severe hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), in patients with renal or pulmonary dysfunction following hematopoietic stem cell transplantation (HSCT).
Enhances the enzymatic activity of plasmin to hydrolyze fibrin clots, though the exact mechanism is not fully elucidated. Studies suggest it may have protective effects on endothelial cells.
Administered as an intravenous infusion. Associated with risk of bleeding and hemorrhage. Monitor for hypotension during infusion. Contraindicated in patients with known hypersensitivity. Safety in pregnancy has not been established.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy