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

 Yun Young Roh  ;  Seung Min Hahn  ;  Hyo Sun Kim  ;  Won Kee Ahn  ;  Jung Hwa Han  ;  Seungyeon Kwon  ;  Chuhl Joo Lyu  ;  Jung Woo Han 
 BONE MARROW TRANSPLANTATION, Vol.56(2) : 411-418, 2021-02 
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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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1. College of Medicine (의과대학) > Others (기타) > 1. Journal Papers
Yonsei Authors
Ahn, Won Kee(안원기) ORCID logo https://orcid.org/0000-0003-3668-7396
Lyu, Chuhl Joo(유철주) ORCID logo https://orcid.org/0000-0001-7124-7818
Hahn, Seung Min(한승민) ORCID logo https://orcid.org/0000-0001-9832-6380
Han, Jung Woo(한정우) ORCID logo https://orcid.org/0000-0001-8936-1205
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