[Defibrotide therapy for patients with sinusoidal obstruction syndrome after hematopoietic stem cell transplantation].
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.
Yakushijin
Kimikazu
K
Hematology, Department of Medicine, Kobe University Graduate School of Medicine.
Okamura
Atsuo
A
Ono
Kanako
K
Kawano
Yuko
Y
Kawano
Hiroki
H
Funakoshi
Yohei
Y
Kawamori
Yuriko
Y
Nishikawa
Shinichiro
S
Minagawa
Kentaro
K
Sada
Akiko
A
Shimoyama
Manabu
M
Yamamoto
Katsuya
K
Katayama
Yoshio
Y
Matsui
Toshimitsu
T
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
D005343:Fibrinolytic Agents; D011089:Polydeoxyribonucleotides; C036901:defibrotide