Successful Treatment with Defibrotide for Sinusoidal Obstruction Syndrome after Hematopoietic Stem Cell Transplantation

概要

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Sinusoidal obstruction syndrome (SOS) (formerly known as hepatic veno-occlusivedisease (VOD)) is a life-threatening complication subsequent to hematopoietic stem celltransplantation. However, no completely satisfactory strategies for the treatment ofSOS have been established yet. Defibrotide is a single-strandedpolydeoxyribonucleotide with anti-thrombotic, anti-ischemic, anti-inflammatory andthrombolytic properties, but without systemic anticoagulant effects, and someencouraging results have been reported in western countries. We treated fourpatients with defibrotide for SOS, since there seemed to be no possibility to cure thepatients with conventionally available treatments in Japan. All patients showedevidence of multiple organ failure at the start of the treatment. Defibrotide wasadministered intravenously in normal saline in four divided doses for 14 to 27 days.Three patients (75%) responded to the therapy, while one died of SOS andcytomegalovirus infection despite intensive therapy. None of the patients sufferedfrom significant adverse effects such as severe hemorrhage. This is the first reportdealing with the treatment with defibrotide of Japanese patients with SOS. Becausedefibrotide is considered to be promising for the treatment of SOS, it is important tostart a phase II study as soon as possible.
神戸大学の論文