Defibrotide — Drug Monograph

Brand names: Defitelio

Drug class: Supportive Care Agent

Mechanism of Action

A mixture of single-stranded polydeoxyribonucleotides that protects and stabilizes endothelial cells. It has profibrinolytic and antithrombotic properties — increasing tissue plasminogen activator and thrombomodulin expression while decreasing plasminogen activator inhibitor-1 and von Willebrand factor — thereby restoring the thrombotic-fibrinolytic balance and reducing the endothelial injury that drives hepatic veno-occlusive disease (sinusoidal obstruction syndrome).

FDA Indications

• Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem cell transplantation (HSCT) (FDA approved 2016)

Common Side Effects

• Hypotension
• Diarrhea
• Vomiting
• Nausea
• Epistaxis

Clinical Pearl

Defibrotide is the only FDA-approved therapy for severe hepatic VOD/SOS after stem cell transplantation. Because it is endothelial-protective and profibrinolytic, bleeding is its defining hazard — avoid concurrent anticoagulants or fibrinolytics and hold it around invasive procedures.

Related Therapies & Mechanisms

• Avatrombopag (Doptelet) — Supportive Care Agent · Supportive Care
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• Eltrombopag (Promacta) — Supportive Care Agent · Supportive Care
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