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Defibrotide

Defibrotide structure

Defibrotide 

structure

Description

Defibrotide is a complex mixture of single stranded polydeoxyribonucleotides derived from porcine intestinal mucosa that has antithrombotic and profibrinolytic activity and is used in the treatment of severe sinusoidal obstruction syndrome (SOS) after hematopoietic cell transplantation (HCT). Defibrotide is used in patients with severe liver injury and has not been associated with worsening of serum aminotransferase elevations during therapy and has not been linked to cases of clinically apparent, idiosyncratic liver injury.

Defibrotide Basic Attributes

444.4

444.11987140

E1942E7K32

B01AX01|B - Blood and blood forming organs

Characteristics

LD50 intraperitoneal in dog: > 600mg/kg

Toxicity

Defibrotide therapy has not been linked to serum aminotransferase elevations or with instances of clinically apparent liver injury separate from the features of SOS for which it is given. In a trial of defibrotide as prophylaxis against SOS conducted in 356 children undergoing HCT, rates of severe adverse events such as hemorrhage, gastrointestinal complaints and liver injury were similar in those receiving defibrotide as in untreated children.

Drug Information

Defibrotide 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.

Defibrotide is a complex mixture of single stranded polydeoxyribonucleotides derived from porcine intestinal mucosa that has antithrombotic and profibrinolytic activity and is used in the treatment of severe sinusoidal obstruction syndrome (SOS) after hematopoietic cell transplantation (HCT). Defibrotide is used in patients with severe liver injury and has not been associated with worsening of serum aminotransferase elevations during therapy and has not been linked to cases of clinically apparent, idiosyncratic liver injury.

Antithrombotic Agents

5-Hydroxy-2-isopropyl-4-(4-(1-methyl-1H-indol-5-yl)-5-oxo-4,5-dihydro-1H-1,2,4-triazol-3-yl)phenyl dihydrogen phosphate

Defibrotide Use and Manufacturing

Human drugs -> Orphan -> Defibrotide -> EMA Drug Category|Antithrombotic agents -> Human pharmacotherapeutic group|Human drugs -> Rare disease (orphan)|Human Drugs -> FDA Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) -> Active Ingredients

Computed Properties

Molecular Weight:444.4
XLogP3:1.8
Hydrogen Bond Donor Count:4
Hydrogen Bond Acceptor Count:7
Rotatable Bond Count:5
Exact Mass:444.11987140
Monoisotopic Mass:444.11987140
Topological Polar Surface Area:137
Heavy Atom Count:31
Complexity:773
Covalently-Bonded Unit Count:1
Compound Is Canonicalized:Yes

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