Drug updated on 12/11/2024
| Dosage Form | Injection (intravenous; 200 mg/2.5 mL [80 mg/mL]) |
| Drug Class | Thrombolytics |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of adult and pediatric patients 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
- This summary is based on the review of six systematic review(s)/meta-analysis(es). [1-6]
- Prophylaxis of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS): Intravenous defibrotide reduced the incidence of VOD/SOS to 5% in adults and 8% in pediatric patients, compared to 16% in controls, with a relative risk of 0.30 (95% confidence interval (CI) 0.12–0.71, p = 0.006).
- Treatment of VOD/SOS with MOD: Defibrotide treatment improved Day 100 survival rates, with a 54% survival rate for patients overall, including 44% for those with multi-organ dysfunction (MOD) and 71% for those without MOD, indicating greater efficacy in patients with less severe disease.
- Efficacy in Severe VOD/SOS Post-Hematopoietic Stem Cell Transplantation (HSCT): In patients with severe VOD post-HSCT, defibrotide demonstrated a Day +100 survival rate of 44% and a complete response rate of 39%, compared to a higher survival rate of 58% and a complete response rate of 57% in the broader population.
- Bleeding Events: The incidence of bleeding events with intravenous defibrotide was 1% (95% CI: 0%-2%) compared to 8% (95% CI: 3%-14%) in controls, with a risk ratio of 0.36 (95% CI: 0.24-0.52; p < .00001), indicating a lower risk of bleeding with defibrotide.
- Adverse Events in Severe VOD/SOS: In patients with severe VOD/SOS, adverse event rates were higher, with 88% experiencing at least one adverse event, 42% reporting hemorrhage, and 58% having serious adverse events (SAEs).
- Both adult and pediatric populations were studied, with a higher incidence of VOD/SOS observed in pediatric patients (8%) compared to adults (5%) when receiving defibrotide prophylaxis. Patients with multi-organ dysfunction (MOD) showed varied survival outcomes, with defibrotide being more effective in patients with less severe MOD (71% survival without MOD vs. 44% with MOD). Additionally, patients post-HSCT with severe VOD exhibited higher adverse event rates, reflecting increased risks within this subgroup.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Defitelio (defibrotide sodium) Prescribing Information. | 2022 | Jazz Pharmaceuticals, Inc., Palo Alto, CA |