Drug updated on 9/4/2024
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).
Latest News
Summary
- Defitelio (defibrotide sodium) 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 (HSCT).
- This summary is based on the review of six systematic review(s)/meta-analysis(es). [1-6]
- Incidence and Risk of VOD/SOS with Defibrotide Prophylaxis: A meta-analysis of 20 studies (N = 3005) reported an overall VOD/SOS incidence of 5% with intravenous defibrotide, with incidences of 5% in adults and 8% in pediatric patients. The risk ratio for developing VOD/SOS with defibrotide prophylaxis compared to controls was 0.30 (95% CI: 0.12-0.71; p = 0.006).
- Day 100 Survival Rates: A meta-analysis of 17 studies (N = 2598) reported an overall Day +100 survival rate of 54%, with 56% for patients treated with ~25 mg/kg/day defibrotide. For patients with MOD, the Day +100 survival was 44%, and 71% in patients without MOD.
- Complete Response and Survival in Severe VOD (sVOD): A meta-analysis of 16 studies (N = 3002) found a Day +100 survival rate of 58% and a CR rate of 57%. In patients with severe VOD, the Day +100 survival rate was 44%, and the CR rate was 39%.
- A meta-analysis of 23 studies reported an estimated overall incidence of bleeding events at 1% (95% CI: 0%-2%) with intravenous defibrotide, compared to 8% (95% CI: 3%-14%) in control groups. The risk ratio for bleeding events with intravenous defibrotide versus controls was 0.36 (95% CI: 0.24-0.52; p < .00001).
- In the VOD/SOS post-HSCT setting, a meta-analysis of 16 studies reported overall adverse events in 65% of patients, hemorrhage in 16%, and serious adverse events in 53%. In patients with severe VOD, the rates were 88% for any adverse event, 42% for hemorrhage, and 58% for serious adverse events.
- Pediatric patients had a higher incidence of VOD/SOS (8%) compared to adults (5%) with intravenous defibrotide prophylaxis; defibrotide-treated patients with less severe MOD had higher Day 100 survival rates than those with more severe MOD; patients with severe VOD had lower survival and complete response rates compared to general VOD/SOS patients; antiplatelet therapy likely increases the risk of bleeding in CKD populations.
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 |