The U.S. Food and Drug Administration (FDA) has approved defibrotide sodium (Defitelio) for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), or sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic cell transplantation (HCT).1
Approval was based on findings from 2 prospective clinical trials and an expanded access study that included a total of 528 patients. Patients with hepatic VOD and multi-organ dysfunction after transplantation received defibrotide sodium 6.25 mg/kg intravenously every 6 hours until resolution of VOD.
Results from study 1, study 2, and study 3 showed that day +100 survival rates were 38% (95% CI, 29 – 48), 44% (95% CI, 33 – 55), and 45% (95% CI, 40 – 51), respectively.
Historical controls with hepatic VOD with renal or pulmonary dysfunction who received supportive care or interventions other than defibrotide sodium had day +100 survival rates ranging from only 21% to 31%.
Clinicians should be aware that defibrotide sodium is contraindicated in patients also receiving systemic anticoagulant or fibrinolytic therapy, and in those with a known hypersensitivity to the drug or to any of its components.
Patients receiving defibrotide sodium should be monitored for bleeding and hypersensitivity reactions. The most common adverse events associated with defibrotide sodium therapy were hypotension, diarrhea, vomiting, nausea, and epistaxis.
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Defibrotide sodium is approved to be administered at a dose of 6.25 mg/kg every 6 hours intravenously over 2 hours for a minimum of 21 days. If signs and symptoms of VOD have not resolved after 21 days, treatment should be continued until resolution.
- Defitelio (defibrotide sodium) [press release]. Silver Springs, MD: U.S. Food and Drug Administration; March 30, 2016. http://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm493278.htm. Accessed March 30, 2016.