Defibrotide sodium

(Defitelio®)

Defibrotide sodium

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Drug updated on 4/26/2024

Dosage FormInjection (intravenous; 200 mg/2.5 mL)
Drug ClassOligonucleotides
Ongoing and
Completed Studies
ClinicalTrials.gov

Indication

  • Indicated for the treatment of adults and children with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT).

Summary
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  • Defibrotide sodium (Defitelio) is indicated for the treatment of adults and children with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation.
  • Six studies were reviewed, comparing Defitelio to other agents in terms of efficacy, safety, and specific considerations across various populations.
  • Unlike traditional agents that primarily focus on reducing myocardial infarction but have associated risks of major and minor bleeding especially in individuals with chronic kidney disease, Defitelio presents a different therapeutic profile being specifically indicated for VOD/SOS.
  • The overall incidence of VOD/SOS was reported at 5% when using intravenous Defitelio prophylaxis compared to a higher relative risk seen in controls. This suggests significant effectiveness particularly among high-risk populations undergoing HSCT differentiated by age groups; a 5% incidence rate observed among adults versus an 8% rate amongst pediatric patients.
  • Despite warnings about potential increases in bleeding risk, one study highlighted an estimated overall incidence of only 1% for such events during studies outside the post-HCT VOD/SOS setting, indicating a lower-than-expected risk compared to control groups, presenting it as safer than traditional agents concerning this context.
  • Patients suffering from varying severities of multi-organ dysfunction post-HCT showed benefits from using Defitelio treatment, including higher Day 100 survival rates observed particularly among those experiencing less severe forms of multi-organ dysfunction, demonstrating its effectiveness across a spectrum of severity levels, although those more severely affected still benefited from its use.

Product Monograph / Prescribing Information

Document TitleYearSource
Defitelio (defibrotide sodium) Prescribing Information.2022Jazz Pharmaceuticals, Inc. Palo Alto, CA

Systematic Reviews / Meta-Analyses

Clinical Practice Guidelines