Prophylactic fresh frozen plasma may prevent development of hepatic VOD after stem cell transplantation via ADAMTS13-mediated restoration of von Willebrand factor plasma levels
Prophylactic fresh frozen plasma may prevent development of hepatic VOD after stem cell transplantation via ADAMTS13-mediated restoration of von Willebrand factor plasma levels
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DOI：
10.1038/sj.bmt.1705724
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发表时间：
2007-08-01
影响因子：
4.8
通讯作者：
Fujimura, Y.
中科院分区：
文献类型：
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作者：
Matsumoto, M.;Kawa, K.;Fujimura, Y.
我们首先进行了一项多中心随机试验（n = 43），随后对参与医院进行了一项问卷调查（n = 209），以评估输注新鲜冷冻血浆（FFP）是否可以预防干细胞移植（SCT）后肝静脉闭塞病（VOD）的发生。43例患者分为两组：23例接受FFP输注，20例未接受FFP输注，其中3例未接受FFP输注的患者发生VOD。血浆血管性血友病因子（VWF）抗原水平较低，在第0天，第7和第28天后，在SCT的患者接受FFP比那些没有接受它，而血浆ADAMTS 13活性（ADAMTS 13：AC）之间没有差异。血浆VWF多聚体（VWFM）被证明是缺陷的高类似中间VWFM在早期SCT后阶段，但有一个显着增加，高VWFM就在VOD发病前。这表明，相对酶底物（ADAMTS 13/高VWFM）的不平衡参与VOD的发病机制。为了加强这一假设，在随机试验中，接受FFP输注的患者VOD的发生率明显低于未接受FFP输注的患者（0/23 vs 3/20）。此外，结合随后的问卷调查研究（0/36 vs 11/173）的结果明确显示发生率具有统计学显著性（0/59 vs 14/193，P = 0.033）。
We initially conducted a multicenter, randomized trial (n = 43), and subsequently a questionnaire study ( n 209) of participating hospitals, to evaluate whether infused fresh frozen plasma (FFP) could prevent the occurrence of hepatic veno-occlusive disease (VOD) after stem cell transplantation (SCT). Forty-three patients were divided into two groups: 23 receiving FFP infusions and 20 not receiving it. VOD developed in three patients not receiving FFP. Plasma von Willebrand factor (VWF) antigen levels were lower at days 0, 7 and 28 after SCT in patients receiving FFP than in those not receiving it, whereas plasma ADAMTS13 activity (ADAMTS13:AC) did not differ between them. Plasma VWF multimer (VWFM) was demonstrated to be defective in the high similar to intermediate VWFM during the early post-SCT phase, but there was a significant increase in high VWFM just before VOD onset. This suggests that a relative enzyme-to-substrate (ADAMTS13/high-VWFM) imbalance is involved in the pathogenesis of VOD. To strengthen this hypothesis, the incidence of VOD was apparently lower in patients receiving FFP infusions than in those not receiving it (0/23 vs 3/20) in the randomized trial. Further, the results combined with the subsequent questionnaire study ( 0/36 vs 11/173) clearly showed the incidence to be statistically significant ( 0/59 vs 14/193, P = 0.033).