Drug updated on 4/24/2024
| Dosage Form | Injection (intravenous; 200 mg/2.5 mL) |
| Drug Class | Oligonucleotides |
| 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
- 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 Title | Year | Source |
|---|---|---|
| Defitelio (defibrotide sodium) Prescribing Information. | 2022 | Jazz Pharmaceuticals, Inc. Palo Alto, CA |
Systematic Reviews / Meta-Analyses
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| BCSH/BSBMT guideline: Diagnosis and management of veno-occlusive disease (sinusoidal obstruction syndrome) following haematopoietic stem cell transplantation. | 2023 | British Journal of Haematology |
| Veno-occlusive disease in HSCT patients: Consensus-based recommendations for risk assessment, diagnosis, and management by the GITMO group. | 2021 | Transplantation |
| A clinician’s guide to diagnosing VOD and treating with defitelio. | 2020 | MD Anderson |