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Defibrotide for Veno-Occlusive Disease Evaluated
Biol Blood Marrow Transplant; ePub 2017 Mar 8; Richardson, et al
Defibrotide showed potential in treating people with veno-occlusive disease or sinusoidal obstruction syndrome, even those with multi-organ dysfunction, according to a treatment protocol study involving 573 individuals.
Investigators administered IV defibrotide at 6.25 mg/kg every 6 hours for 21 days. The following patients were alive 100 or more days after receiving HSCT:
- Half of the overall study population.
- 55% of patients ≤16 years of age.
- 45% in those >16 years of age.
- 46% of patients with multi-organ dysfunction.
Adverse events occurred in ~7 in every 10 patients; the most common treatment-related adverse event was hypotension, which was seen in ~14%.
Richardson P, Smith A, Triplett B, et al. Defibrotide for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS): Interim results from a treatment IND study. [Published online ahead of print March 8, 2017]. Biol Blood Marrow Transplant. doi:10.1016/j.bbmt.2017.03.008.