Defibrotide (Defitelio)
General information
Class/mechanism: Profibrinolytic. Defibrotide is a mixture of polydeoxyribonucleotide sodium salts made from porcine intestinal tissue. In vitro, it boosts enzymatic activity of plasmin to hydrolyze fibrin clots. Defibrotide increases tissue plasminogen activator (t-PA) and thrombomodulin expression while decreasing von Willebrand factor (vWF) and plasminogen activator inhibitor-1 (PAI-1) expression. Endothelial cell fibrinolysis is enchanced and endothelial cell activation is reduced. Defibrotide appears to limit damage caused by chemotherapy, tumor necrosis factor-α (TNF-α), serum starvation, and perfusion.[1][2][3]
Route: IV
Extravasation: no information
For conciseness and simplicity, HemOnc.org currently will focus on treatment regimens and not list information such as: renal/hepatic dose adjustments, metabolism (including CYP450), excretion, monitoring parameters (although this will be considered for checklists), or manufacturer. Instead, for the most current information, please refer to your preferred pharmacopeias such as Micromedex, Lexicomp, UpToDate (courtesy of Lexicomp), or the prescribing information.[1]
Diseases for which it is used
- Hepatic veno-occlusive disease (VOD), sinusoidal obstructive syndrome (SOS) with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT)
Patient drug information
- Brief patient counseling information available in the Defibrotide (Defitelio) package insert[1]
History of changes in FDA indication
- 2016-03-30: FDA approved "for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstructive syndrome, with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT)."
History of changes in EMA indication
- 2013-10-18: Initial authorization
- Uncertain date: Defitelio is indicated for the treatment of severe hepatic veno-occlusive disease (VOD) also known as sinusoidal obstructive syndrome (SOS) in haematopoietic stem-cell transplantation (HSCT) therapy. It is indicated in adults and in adolescents, children and infants over 1 month of age.
History of changes in PMDA indication
- 2019-06-18: Newly indicated for the treatment of sinusoidal obstruction syndrome (SOS) /hepatic veno-occlusive disease (VOD).
Also known as
- Generic name: polydeoxyribonucleotide sodium salt
- Brand name: Defitelio