title: [Defibrotide therapy for patients with sinusoidal obstruction syndrome after hematopoietic stem cell transplantation].
abstract: Sinusoidal obstruction syndrome (SOS) is one of the life-threatening complications caused by endothelial damage to the hepatic sinusoids after hematopoietic stem cell transplantation. However, a satisfactory treatment for SOS has not yet been established. Defibrotide has anti-thrombotic, anti-ischemic, anti-inflammatory, and thrombolytic properties without systemic anticoagulant effects. We treated eight post-transplant SOS patients with defibrotide. Three patients responded to the therapy and the initial response was observed within a week. In addition to the improvement of liver function, rapid recovery of response to diuretic drugs followed by the improvement of renal function was observed. All of the five patients with respiratory dysfunction died despite administration of defibrotide, suggesting that early treatment might lead to better outcomes. There were no severe adverse effects directly due to defibrotide administration. Defibrotide seems to be a promising treatment for SOS, and the initiation of a clinical study in Japan would be important.
authors.affiliation: Hematology, Department of Medicine, Kobe University Graduate School of Medicine.
authors.forename: Kimikazu
authors.initials: K
authors.lastname: Yakushijin
authors.forename: Atsuo
authors.initials: A
authors.lastname: Okamura
authors.forename: Kanako
authors.initials: K
authors.lastname: Ono
authors.forename: Yuko
authors.initials: Y
authors.lastname: Kawano
authors.forename: Hiroki
authors.initials: H
authors.lastname: Kawano
authors.forename: Yohei
authors.initials: Y
authors.lastname: Funakoshi
authors.forename: Yuriko
authors.initials: Y
authors.lastname: Kawamori
authors.forename: Shinichiro
authors.initials: S
authors.lastname: Nishikawa
authors.forename: Kentaro
authors.initials: K
authors.lastname: Minagawa
authors.forename: Akiko
authors.initials: A
authors.lastname: Sada
authors.forename: Manabu
authors.initials: M
authors.lastname: Shimoyama
authors.forename: Katsuya
authors.initials: K
authors.lastname: Yamamoto
authors.forename: Yoshio
authors.initials: Y
authors.lastname: Katayama
authors.forename: Toshimitsu
authors.initials: T
authors.lastname: Matsui
mesh_terms: D000293:Adolescent; D000328:Adult; D005260:Female; D005343:Fibrinolytic Agents / Q000008:administration & dosage*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006504:Hepatic Veno-Occlusive Disease / Q000188:drug therapy* / Q000209:etiology*; D006801:Humans; D008297:Male; D008875:Middle Aged; D011089:Polydeoxyribonucleotides / Q000008:administration & dosage*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D005343:Fibrinolytic Agents; D011089:Polydeoxyribonucleotides; C036901:defibrotide