The use of recombinant factor VIIa in children with inherited platelet function disorders
The use of recombinant factor VIIa in children with inherited platelet function disorders
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DOI：
10.1046/j.1365-2141.2003.04286.x
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发表时间：
2003-05-01
影响因子：
6.5
通讯作者：
Liesner, R
中科院分区：
文献类型：
--
作者：
Almeida, AM;Khair, K;Liesner, R
遗传性血小板表面糖蛋白缺陷，如Glanzmann血栓减少症（GT）或Bernard-Soulier综合征（BSS）可导致严重的出血性素质。在过去，这些患者的出血发作通常需要输注血小板以确保止血，但最近一些患者报告表明重组因子VIIa （rVIIa）也可能有效。在2年的时间里，我们对7名遗传性血小板功能障碍的儿童进行了33次rVIIa治疗，其中5名患有GT， 1名患有BSS， 1名患有严重表型的储存池病。在28例急性出血发作中，仅rVIIa就有10例出血停止，但其中2例复发。预测对rVIIa的反应的两个特征是出血的严重程度和从出血到治疗的延迟。rVIIa成功治疗了5例计划手术干预。28例急性发作中有18例和没有一例计划手术发作需要血液制品支持。我们发现rVIIa治疗遗传性血小板功能缺陷儿童急性出血发作的疗效不同，但更大规模的研究是有必要的，特别是rVIIa是一种相对低风险的治疗方法。
Inherited deficiencies of platelet surface glycoproteins such as Glanzmann's thrombasthenia (GT) or Bernard-Soulier syndrome (BSS) can lead to a severe bleeding diathesis. In the past, bleeding episodes in these patients have often required platelet transfusion to secure haemostasis but recently a number of patient reports have suggested that recombinant factor VIIa (rVIIa) may also be effective. We have used rVIIa on 33 occasions in seven children with inherited platelet function disorders over a 2-year period: five had GT, one had BSS and one had storage pool disease with a severe phenotype. Bleeding ceased with rVIIa alone in 10 of 28 acute bleeding episodes, but recurred in two of these. The two features that predicted response to rVIIa were the severity of the bleeding and the delay from the onset of bleeding to treatment. Five episodes of planned surgical intervention were treated successfully with rVIIa. Eighteen out of the 28 acute episodes and none of the planned surgical episodes required blood product support. We have found variable efficacy of rVIIa for acute bleeding episodes in this small series of children with inherited platelet function defects but larger studies are warranted, particularly as rVIIa is a relatively low-risk treatment approach for these disorders.