Drug updated on 4/15/2024
| 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.
- A total of 6 systematic reviews/meta-analyses were reviewed to gather information about this drug.
- In a meta-analysis, defibrotide was found to have a low incidence of VOD/SOS following prophylaxis, regardless of age group, and showed lower relative risk for VOD/SOS compared to controls in patient populations at high risk of VOD/SOS.
- Another study suggested that the incidence of bleeding events with defibrotide use outside post-hematopoietic cell transplantation setting is lower than controls which indicates its safety profile regarding bleeding risks.
- Pooled analysis from three studies demonstrated higher Day 100 survival rates amongst patients treated with Defitelio having less severe forms versus more severe forms multi-organ dysfunction after haematopoietic cell transplantation indicating its effectiveness based on severity level.
- However, one review/meta-analysis highlighted that while defibrotide improves day +100 survival rate and complete response in both general VOD/sVOD patients post-HSCT, these results are mainly based on observational data potentially subject to selection bias suggesting need for further randomized control trials and larger prospective cohort studies for validation purposes.
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 |