Clinical effects of recombinant thrombomodulin and defibrotide on sinusoidal obstruction syndrome after allogeneic hematopoietic stem cell transplantation

Kimikazu Yakushijin, Takayuki Ikezoe, Chikako Ohwada, Kazuko Kudo, Hiroshi Okamura, Hiroaki Goto, Hiromasa Yabe, Atsushi Yasumoto, Hideyuki Kuwabara, Shiro Fujii, Kumiko Kagawa, Masao Ogata, Yasushi Onishi, Akio Kohno, Koichi Watamoto, Nobuhiko Uoshima, Daisuke Nakamura, Shuichi Ota, Yasunori Ueda, Tatsuo Oyake & 9 others Kazutoshi Koike, Ishikazu Mizuno, Hiroatsu Iida, Yoshio Katayama, Hiroatsu Ago, Koji Kato, Atsuo Okamura, Atsushi Kikuta, Takahiro Fukuda

研究成果: ジャーナルへの寄稿記事

1 引用 (Scopus)

抄録

Sinusoidal obstruction syndrome (SOS) is a lethal complication after hematopoietic stem cell transplantation (HSCT). Defibrotide (DF) is the only drug internationally recommended for SOS treatment in Western countries. Recombinant human soluble thrombomodulin (rhTM), which is promising for the treatment of patients with disseminated intravascular coagulation, is also reported to be potentially effective for SOS. To clarify the safety and efficacy of DF and rhTM, we conducted a retrospective survey of these agents in Japan. Data from 65 patients who underwent allogeneic HSCT and received DF (n = 24) or rhTM (n = 41) for SOS treatment were collected. The complete response rates for SOS on day 100 were 50% and 54% in the DF and rhTM groups, respectively. The 100-day overall survival rates were 50% in the DF group, and 48% in the rhTM group. Several severe hemorrhagic adverse events were observed in one patient in the DF group and five patients in the rhTM group. The main causes of death were SOS-related death, and no patient died of direct adverse events of DF or rhTM. Our results suggest that rhTM, as well as DF, can be effective as a novel treatment option for SOS.

元の言語英語
ページ(範囲)674-680
ページ数7
ジャーナルBone Marrow Transplantation
54
発行部数5
DOI
出版物ステータス出版済み - 5 1 2019

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Hepatic Veno-Occlusive Disease
Thrombomodulin
Hematopoietic Stem Cell Transplantation
human THBD protein
defibrotide
Disseminated Intravascular Coagulation
Therapeutics
Cause of Death
Japan
Survival Rate
Safety

All Science Journal Classification (ASJC) codes

  • Hematology
  • Transplantation

これを引用

Clinical effects of recombinant thrombomodulin and defibrotide on sinusoidal obstruction syndrome after allogeneic hematopoietic stem cell transplantation. / Yakushijin, Kimikazu; Ikezoe, Takayuki; Ohwada, Chikako; Kudo, Kazuko; Okamura, Hiroshi; Goto, Hiroaki; Yabe, Hiromasa; Yasumoto, Atsushi; Kuwabara, Hideyuki; Fujii, Shiro; Kagawa, Kumiko; Ogata, Masao; Onishi, Yasushi; Kohno, Akio; Watamoto, Koichi; Uoshima, Nobuhiko; Nakamura, Daisuke; Ota, Shuichi; Ueda, Yasunori; Oyake, Tatsuo; Koike, Kazutoshi; Mizuno, Ishikazu; Iida, Hiroatsu; Katayama, Yoshio; Ago, Hiroatsu; Kato, Koji; Okamura, Atsuo; Kikuta, Atsushi; Fukuda, Takahiro.

:: Bone Marrow Transplantation, 巻 54, 番号 5, 01.05.2019, p. 674-680.

研究成果: ジャーナルへの寄稿記事

Yakushijin, K, Ikezoe, T, Ohwada, C, Kudo, K, Okamura, H, Goto, H, Yabe, H, Yasumoto, A, Kuwabara, H, Fujii, S, Kagawa, K, Ogata, M, Onishi, Y, Kohno, A, Watamoto, K, Uoshima, N, Nakamura, D, Ota, S, Ueda, Y, Oyake, T, Koike, K, Mizuno, I, Iida, H, Katayama, Y, Ago, H, Kato, K, Okamura, A, Kikuta, A & Fukuda, T 2019, 'Clinical effects of recombinant thrombomodulin and defibrotide on sinusoidal obstruction syndrome after allogeneic hematopoietic stem cell transplantation', Bone Marrow Transplantation, 巻. 54, 番号 5, pp. 674-680. https://doi.org/10.1038/s41409-018-0304-4
Yakushijin, Kimikazu ; Ikezoe, Takayuki ; Ohwada, Chikako ; Kudo, Kazuko ; Okamura, Hiroshi ; Goto, Hiroaki ; Yabe, Hiromasa ; Yasumoto, Atsushi ; Kuwabara, Hideyuki ; Fujii, Shiro ; Kagawa, Kumiko ; Ogata, Masao ; Onishi, Yasushi ; Kohno, Akio ; Watamoto, Koichi ; Uoshima, Nobuhiko ; Nakamura, Daisuke ; Ota, Shuichi ; Ueda, Yasunori ; Oyake, Tatsuo ; Koike, Kazutoshi ; Mizuno, Ishikazu ; Iida, Hiroatsu ; Katayama, Yoshio ; Ago, Hiroatsu ; Kato, Koji ; Okamura, Atsuo ; Kikuta, Atsushi ; Fukuda, Takahiro. / Clinical effects of recombinant thrombomodulin and defibrotide on sinusoidal obstruction syndrome after allogeneic hematopoietic stem cell transplantation. :: Bone Marrow Transplantation. 2019 ; 巻 54, 番号 5. pp. 674-680.
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abstract = "Sinusoidal obstruction syndrome (SOS) is a lethal complication after hematopoietic stem cell transplantation (HSCT). Defibrotide (DF) is the only drug internationally recommended for SOS treatment in Western countries. Recombinant human soluble thrombomodulin (rhTM), which is promising for the treatment of patients with disseminated intravascular coagulation, is also reported to be potentially effective for SOS. To clarify the safety and efficacy of DF and rhTM, we conducted a retrospective survey of these agents in Japan. Data from 65 patients who underwent allogeneic HSCT and received DF (n = 24) or rhTM (n = 41) for SOS treatment were collected. The complete response rates for SOS on day 100 were 50{\%} and 54{\%} in the DF and rhTM groups, respectively. The 100-day overall survival rates were 50{\%} in the DF group, and 48{\%} in the rhTM group. Several severe hemorrhagic adverse events were observed in one patient in the DF group and five patients in the rhTM group. The main causes of death were SOS-related death, and no patient died of direct adverse events of DF or rhTM. Our results suggest that rhTM, as well as DF, can be effective as a novel treatment option for SOS.",
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T1 - Clinical effects of recombinant thrombomodulin and defibrotide on sinusoidal obstruction syndrome after allogeneic hematopoietic stem cell transplantation

AU - Yakushijin, Kimikazu

AU - Ikezoe, Takayuki

AU - Ohwada, Chikako

AU - Kudo, Kazuko

AU - Okamura, Hiroshi

AU - Goto, Hiroaki

AU - Yabe, Hiromasa

AU - Yasumoto, Atsushi

AU - Kuwabara, Hideyuki

AU - Fujii, Shiro

AU - Kagawa, Kumiko

AU - Ogata, Masao

AU - Onishi, Yasushi

AU - Kohno, Akio

AU - Watamoto, Koichi

AU - Uoshima, Nobuhiko

AU - Nakamura, Daisuke

AU - Ota, Shuichi

AU - Ueda, Yasunori

AU - Oyake, Tatsuo

AU - Koike, Kazutoshi

AU - Mizuno, Ishikazu

AU - Iida, Hiroatsu

AU - Katayama, Yoshio

AU - Ago, Hiroatsu

AU - Kato, Koji

AU - Okamura, Atsuo

AU - Kikuta, Atsushi

AU - Fukuda, Takahiro

PY - 2019/5/1

Y1 - 2019/5/1

N2 - Sinusoidal obstruction syndrome (SOS) is a lethal complication after hematopoietic stem cell transplantation (HSCT). Defibrotide (DF) is the only drug internationally recommended for SOS treatment in Western countries. Recombinant human soluble thrombomodulin (rhTM), which is promising for the treatment of patients with disseminated intravascular coagulation, is also reported to be potentially effective for SOS. To clarify the safety and efficacy of DF and rhTM, we conducted a retrospective survey of these agents in Japan. Data from 65 patients who underwent allogeneic HSCT and received DF (n = 24) or rhTM (n = 41) for SOS treatment were collected. The complete response rates for SOS on day 100 were 50% and 54% in the DF and rhTM groups, respectively. The 100-day overall survival rates were 50% in the DF group, and 48% in the rhTM group. Several severe hemorrhagic adverse events were observed in one patient in the DF group and five patients in the rhTM group. The main causes of death were SOS-related death, and no patient died of direct adverse events of DF or rhTM. Our results suggest that rhTM, as well as DF, can be effective as a novel treatment option for SOS.

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