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

Research output: Contribution to journalArticle

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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 1 2009

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Hepatic Veno-Occlusive Disease
Hematopoietic Stem Cell Transplantation
Therapeutics
Liver
Recovery of Function
Diuretics
Anticoagulants
Japan
Anti-Inflammatory Agents
defibrotide
Transplants
Kidney
Pharmaceutical Preparations

All Science Journal Classification (ASJC) codes

  • Medicine(all)

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Yakushijin, Kimikazu ; Okamura, Atsuo ; Ono, Kanako ; Kawano, Yuko ; Kawano, Hiroki ; Funakoshi, Yohei ; Kawamori, Yuriko ; Nishikawa, Shinichiro ; Minagawa, Kentaro ; Sada, Akiko ; Shimoyama, Manabu ; Yamamoto, Katsuya ; Katayama, Yoshio ; Matsui, Toshimitsu. / Defibrotide therapy for patients with sinusoidal obstruction syndrome after hematopoietic stem cell transplantation. In: [Rinshō ketsueki] The Japanese journal of clinical hematology. 2009 ; Vol. 50, No. 1. pp. 3-8.
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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.",
author = "Kimikazu Yakushijin and Atsuo Okamura and Kanako Ono and Yuko Kawano and Hiroki Kawano and Yohei Funakoshi and Yuriko Kawamori and Shinichiro Nishikawa and Kentaro Minagawa and Akiko Sada and Manabu Shimoyama and Katsuya Yamamoto and Yoshio Katayama and Toshimitsu Matsui",
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Yakushijin, K, Okamura, A, Ono, K, Kawano, Y, Kawano, H, Funakoshi, Y, Kawamori, Y, Nishikawa, S, Minagawa, K, Sada, A, Shimoyama, M, Yamamoto, K, Katayama, Y & Matsui, T 2009, 'Defibrotide therapy for patients with sinusoidal obstruction syndrome after hematopoietic stem cell transplantation', [Rinshō ketsueki] The Japanese journal of clinical hematology, vol. 50, no. 1, pp. 3-8.

Defibrotide therapy for patients with sinusoidal obstruction syndrome after hematopoietic stem cell transplantation. / Yakushijin, Kimikazu; Okamura, Atsuo; Ono, Kanako; Kawano, Yuko; Kawano, Hiroki; Funakoshi, Yohei; Kawamori, Yuriko; Nishikawa, Shinichiro; Minagawa, Kentaro; Sada, Akiko; Shimoyama, Manabu; Yamamoto, Katsuya; Katayama, Yoshio; Matsui, Toshimitsu.

In: [Rinshō ketsueki] The Japanese journal of clinical hematology, Vol. 50, No. 1, 01.01.2009, p. 3-8.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Defibrotide therapy for patients with sinusoidal obstruction syndrome after hematopoietic stem cell transplantation

AU - Yakushijin, Kimikazu

AU - Okamura, Atsuo

AU - Ono, Kanako

AU - Kawano, Yuko

AU - Kawano, Hiroki

AU - Funakoshi, Yohei

AU - Kawamori, Yuriko

AU - Nishikawa, Shinichiro

AU - Minagawa, Kentaro

AU - Sada, Akiko

AU - Shimoyama, Manabu

AU - Yamamoto, Katsuya

AU - Katayama, Yoshio

AU - Matsui, Toshimitsu

PY - 2009/1/1

Y1 - 2009/1/1

N2 - 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.

AB - 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.

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