title: Successful treatment with defibrotide for sinusoidal obstruction syndrome after hematopoietic stem cell transplantation.
abstract: Sinusoidal obstruction syndrome (SOS) (formerly known as hepatic veno-occlusive disease (VOD)) is a life-threatening complication subsequent to hematopoietic stem cell transplantation. However, no completely satisfactory strategies for the treatment of SOS have been established yet. Defibrotide is a single-stranded polydeoxyribonucleotide with anti-thrombotic, anti-ischemic, anti-inflammatory and thrombolytic properties, but without systemic anticoagulant effects, and some encouraging results have been reported in western countries. We treated four patients with defibrotide for SOS, since there seemed to be no possibility to cure the patients with conventionally available treatments in Japan. All patients showed evidence of multiple organ failure at the start of the treatment. Defibrotide was administered 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 and cytomegalovirus infection despite intensive therapy. None of the patients suffered from significant adverse effects such as severe hemorrhage. This is the first report dealing with the treatment with defibrotide of Japanese patients with SOS. Because defibrotide is considered to be promising for the treatment of SOS, it is important to start a phase II study as soon as possible.
authors.affiliation: Division of Endocrinology/Metabolism, Neurology and Hematology/Oncology, Department of Clinical Molecular Medicine, Kobe University Graduate School of Medicine. kyakushi@med.kobe.u-ac.jp
authors.forename: Kimikazu
authors.initials: K
authors.lastname: Yakushijin
authors.forename: Toshimitsu
authors.initials: T
authors.lastname: Matsui
authors.forename: Atsuo
authors.initials: A
authors.lastname: Okamura
authors.forename: Katsuya
authors.initials: K
authors.lastname: Yamamoto
authors.forename: Mitsuhiro
authors.initials: M
authors.lastname: Ito
authors.forename: Kazuo
authors.initials: K
authors.lastname: Chihara
mesh_terms: D000328:Adult; D000990:Antithrombin III / Q000627:therapeutic use; D005260:Female; D005343:Fibrinolytic Agents / Q000627:therapeutic use*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006504:Hepatic Veno-Occlusive Disease / Q000175:diagnosis / Q000188:drug therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D011089:Polydeoxyribonucleotides / Q000627:therapeutic use*; D010959:Tissue Plasminogen Activator / Q000627:therapeutic use; D019172:Transplantation Conditioning / Q000009:adverse effects
chemical_list: D005343:Fibrinolytic Agents; D011089:Polydeoxyribonucleotides; C036901:defibrotide; D000990:Antithrombin III; D010959:Tissue Plasminogen Activator