Defitelio (defibrotide sodium)

Thrombolytic Agents Intravenous Vial (Ml)

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Last reviewed · Source: First Databank (FDB) drug data

Overview

Defibrotide sodium, a polydisperse mixture of predominantly single-stranded polydeoxyribonucleotide sodium salts derived from porcine intestinal tissue, is an antithrombotic agent with anti-inflammatory, anti-ischemic, and thrombolytic properties.

Defibrotide, a polydisperse oligonucleotide mixture of predominantly single-stranded polydeoxyribonucleotide sodium salts derived from porcine intestinal tissue, possesses anti-inflammatory, anti-ischemic, antithrombotic, and thrombolytic properties.

Hepatic veno-occlusive disease (VOD) following hematopoietic stem cell transplantation (HSCT) is caused by sinusoidal endothelial cell damage from radiation and cancer chemotherapy agents used during the HSCT procedure. Endothelial cell damage activates proinflammatory pathways, which results in local cell damage, allows cellular debris to flow into the perisinusoidal space, causes detachment of the sinusoidal lining, and leads to activation of the coagulation pathway. The subsequent activation of the fibrinolytic pathway causes fibrosis of the sinusoids, which leads to perivascular hepatocyte necrosis and blockage of venules in the liver.

These changes result in a reduction of hepatic venous outflow, which causes postsinusoidal hypertension, declining liver function, and eventually multiorgan failure and death. The exact mechanism of action of defibrotide in the treatment of hepatic VOD has not been fully elucidated, but is thought to involve endothelial protection and restoration of the thrombotic-fibrinolytic balance leading to preservation of hepatic sinusoidal blood flow. Defibrotide has been shown to protect endothelial cells from damage by cancer chemotherapy agents, tumor necrosis factor (TNF; TNF-alpha), serum starvation, and perfusion.

Defibrotide also has been shown to reduce endothelial cell activation and increase endothelial cell-mediated fibrinolysis by increasing tissue plasminogen activator (t-PA) and thrombomodulin expression and decreasing von Willebrand factor and plasminogen activator inhibitor-1 (PAI-1) expression. In vitro, defibrotide enhanced the enzymatic activity of plasmin to hydrolyze fibrin clots.

Following IV administration, peak plasma concentrations of defibrotide sodium are reached at the end of each infusion. The elimination half-life of defibrotide sodium is less than 2 hours. In vitro studies have shown that defibrotide sodium does not undergo substantial metabolism by hepatocytes.

While the precise pathway by which defibrotide sodium is degraded in vivo is unknown, it is thought that polynucleotides are metabolized progressively to oligonucleotides, nucleotides, nucleosides, and then to free 2'-deoxyribose sugar (purine and pyrimidine bases) by nucleases, nucleosidases, deaminases, and phosphorylases.

Generic name: defibrotide sodium
Drug class: Thrombolytic Agents
Therapeutic class: Hematological Agents

Reference product & biosimilars

No reference, biosimilar or interchangeable associated with this drug.

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Indications

The following indications are listed for Defitelio:

  • Hepatic Veno-Occlusive Disease

Also known as: Sinusoidal Obstruction Syndrome, Veno-Occlusive Disease Of The Liver.

Dosing

Dosage of defibrotide sodium is expressed in terms of the salt. Defibrotide sodium has a potency of 27-39 units per mg based on results of 2 separate assays that measure the release of a product formed by contact between defibrotide sodium, plasmin, and a plasmin substrate.

Drug interactions

Formal drug interaction studies with defibrotide are lacking.

Defibrotide is not a substrate, inhibitor, or inducer of any known drug-metabolizing enzymes or transporters.

Contraindications

Concomitant administration with systemic anticoagulant or fibrinolytic therapy. (See Hemorrhage under Cautions: Warnings/Precautions.) Known hypersensitivity to defibrotide or any ingredient(s) in the formulation.

Adverse reactions

Hemorrhagic events of any type or severity were reported in 59% of 176 patients receiving defibrotide sodium (6.25 mg/kg every 6 hours) in clinical trials for the treatment of hepatic VOD with pulmonary and/or renal dysfunction following HSCT; 20% of patients experienced grade 4-5 (fatal or considered life-threatening) hemorrhagic events. Adverse effects (independent of causality) reported in at least 10% of patients receiving defibrotide in clinical trials were hypotension, diarrhea, vomiting, nausea, and epistaxis. Life-threatening or fatal adverse events (independent of causality) reported in at least 2% of patients receiving defibrotide in clinical trials include pulmonary alveolar hemorrhage, sepsis, graft-versus-host disease, GI hemorrhage, lung infiltration, pneumonia, pulmonary hemorrhage, infection, intracranial hemorrhage, hyperuricemia, and cerebral hemorrhage.

Precautions

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ICD codes

CodeTypeDescription
Hepatic veno-occlusive disease
K76.5ICD10CMHepatic veno-occlusive disease

ICD code information from First Databank (FDB).

Formulary status

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Last reviewed · Source: First Databank (FDB) drug data