Defibrotide is an effective treatment for sinusoidal obstruction syndrome (SOS), also known as hepatic veno-occlusive disease (VOD), in patients undergoing hematopoietic stem cell transplantation (HSCT), according to a study published in Supportive Care in Cancer.

SOS/VOD, a complication observed in patients who undergo HSCT, is characterized by injury to the hepatic venules and sinusoidal endothelial cells; may lead to symptoms such as elevated bilirubin concentrations, hepatomegaly, jaundice, weight gain, and ascites; and is potentially fatal.

Defibrotide is recommended for nearly all management protocols for SOS/VOD, yet due to its high cost and limited accessibility, studies to determine ideal patient selection, dose, and duration have been limited. The purpose of this study was to assess the efficacy of defibrotide and evaluate response to therapy at guideline-specified time points.

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For this study, researchers retrospectively analyzed data from 23 patients with leukemia who underwent HSCT and were treated with a median dose of defibrotide 25 mg/kg/day. Continuation of defibrotide therapy was contingent upon stabilization or improvement of symptoms or biochemical markers.

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Fourteen (61%) patients responded to defibrotide therapy.

At a median follow-up of approximately 3.7 years, overall survival (OS) was 35%. At day 100 after HSCT, overall survival was 70%.

All evaluable patients experienced a decrease in their median serum (total) bilirubin concentrations at days 5 and 10 (P <.001), and a proportional decrease in median body weight of 4% and 6.6% on days 5 and 10, respectively, was observed.

Patients treated with defibrotide experienced a significant and continued clinical improvement of hepatomegaly (P =.02), ascites (P <.01), and requirement of oxygen supplementation (P <.01) from baseline to day 5, and to day 10.

Defibrotide is an effective treatment for SOS/VOD and should be continued based on the attainment of early response. The authors concluded “[d]efribotide should be considered in any consensus protocol providing guidance on the management of SOS/VOD, with future studies considered to assess appropriate time points for response to therapy during treatment.”


1. Coutsouvelis J, Avery S, Dooley M, Kirkpatrick C, Spencer A. Defibrotide for the treatment of sinusoidal obstruction syndrome: evaluation of response to therapy and patient outcomes [published online October 11, 2017]. Support Care Cancer. doi: 10.1007/s00520-017-3915-1