Defibrotide therapy for patients with sinusoidal obstruction syndrome after hematopoietic stem cell transplantation

Kimikazu Yakushijin, Atsuo Okamura, Kanako Ono, Yuko Kawano, Hiroki Kawano, Yohei Funakoshi, Yuriko Kawamori, Shinichiro Nishikawa, Kentaro Minagawa, Akiko Sada, Manabu Shimoyama, Katsuya Yamamoto, Yoshio Katayama, Toshimitsu Matsui

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1 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)3-8
Number of pages6
Journal[Rinshō ketsueki] The Japanese journal of clinical hematology
Volume50
Issue number1
StatePublished - Jan 2009

All Science Journal Classification (ASJC) codes

  • Medicine(all)

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