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Efficacy of low dose and short duration defibrotide prophylaxis for hepatic veno-occlusive disease after autologous haematopoietic stem cell transplantation

Bone Marrow Transplantation volume 56, pages 411–418 (2021)Cite this article

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Abstract

Hepatic veno-occlusive disease (VOD) is a serious systemic endothelial complication after stem cell transplantation. Defibrotide is under investigation as a prophylactic agent for VOD; however, high costs limit its utility. We evaluated the prophylactic efficacy of a low-dose defibrotide regimen for VOD. We retrospectively enrolled 147 paediatric patients who underwent autologous haematopoietic stem cell transplantation (HSCT; 69 with defibrotide prophylaxis and 78 historical controls) at the Yonsei Cancer Center in Seoul, Korea, between March 2013 and Feb 2020. Low-dose defibrotide (12.5 mg/kg/day) was administered from D−3 to D+10 after HSCT. The most common diagnosis in the cohort was brain tumour (N = 86). VOD developed in 10 (12.8%) and 3 (4.3%) patients in the control and prophylaxis groups, respectively (P = 0.071). In the second HSCT group, VOD incidence was significantly lower in the prophylaxis group [2.9% (1/35)] than in the control group (28.6%, 6/21, P = 0.005). VOD severity was significantly higher in the control group than in the prophylaxis group (P = 0.006). Three VOD-related mortalities occurred in the control group, whereas no VOD-related mortality occurred in the prophylaxis group. In conclusion, low-dose defibrotide prophylaxis is a promising and economical strategy for preventing VOD, especially in second-round HSCT.

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Fig. 1: Cumulative incidence of veno-occlusive disease (VOD).
Fig. 2: Severity of veno-occlusive disease (VOD).

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Authors and Affiliations

  1. Department of Pediatric Hematology-Oncology, Yonsei Cancer Center, Yonsei University Health System, Seoul, Korea

    Yun Young Roh, Seung Min Hahn, Hyo Sun Kim, Won Kee Ahn, Jung Hwa Han, Seungyeon Kwon, Chuhl Joo Lyu & Jung Woo Han

  2. Department of Pediatrics, Yonsei University College of Medicine, Yonsei University Health System, Seoul, Korea

    Yun Young Roh, Seung Min Hahn, Hyo Sun Kim, Won Kee Ahn, Seungyeon Kwon, Chuhl Joo Lyu & Jung Woo Han

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Roh, Y.Y., Hahn, S.M., Kim, H.S. et al. Efficacy of low dose and short duration defibrotide prophylaxis for hepatic veno-occlusive disease after autologous haematopoietic stem cell transplantation. Bone Marrow Transplant 56, 411–418 (2021). https://doi.org/10.1038/s41409-020-01036-5

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