Coagulation factor activity and clinical bleeding severity in rare bleeding disorders: results from the European Network of Rare Bleeding Disorders
Coagulation factor activity and clinical bleeding severity in rare bleeding disorders: results from the European Network of Rare Bleeding Disorders
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DOI：
10.1111/j.1538-7836.2012.04653.x
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发表时间：
2012-04-01
影响因子：
10.4
通讯作者：
Rosendaal, F. R.
中科院分区：
文献类型：
--
作者：
Peyvandi, F.;Palla, R.;Rosendaal, F. R.
背景资料：欧洲罕见出血性疾病网络（EN-RBD）的建立是为了弥合RBD患者护理知识与实践之间的差距。目的：探讨RBD患者凝血因子活性水平与出血严重程度的关系。患者/方法：采用EN-RBD中登记的489例患者的数据进行横断面研究。检索凝血因子活性水平。根据严重程度将临床出血事件分为四类。结果：数据收集时患者的平均年龄为31岁（范围：7个月至95岁），性别分布均匀。在线性回归分析中，凝血因子活性水平与纤维蛋白原、因子（F）X、FXIII以及FV和FVIII联合缺乏的临床出血严重程度之间存在强相关性。一个较弱的关联是FV和FVII缺陷。凝血因子活性水平与FXI的临床出血严重程度之间无相关性。患者保持无症状所需的凝血因子活性水平为：纤维蛋白原，> 100 mg dL（-1）; FV，12 U dL（-1）;合并FV + VIII，43 U dL（-1）; FVII，25 U dL（-1）; FX，56 U dL（-1）; FXI，26 U dL（-1）; FXIII，31 U dL（-1）。此外，对应于III级出血的凝血因子活性水平为：纤维蛋白原、FV和FXIII的不可检测水平，FV + VIII组合< 15 U dL（-1）; FVI < 8 U dL（-1）; FX < 10 U dL（-1）; FXI < 25 U dL（-1）。结论：不同RBD患者凝血因子活性水平与临床出血严重程度之间存在异质性相关。仅在纤维蛋白原、FX和FXIII缺乏中观察到强关联。
Background: The European Network of Rare Bleeding Disorders (EN-RBD) was established to bridge the gap between knowledge and practise in the care of patients with RBDs. Objectives: To explore the relationship between coagulation factor activity level and bleeding severity in patients with RBDs. Patients/Methods: Cross-sectional study using data from 489 patients registered in the EN-RBD. Coagulation factor activity levels were retrieved. Clinical bleeding episodes were classified into four categories according to severity. Results: The mean age of patients at data collection was 31 years (range, 7 months to 95 years), with an equal sex distribution. On linear regression analysis, there was a strong association between coagulation factor activity level and clinical bleeding severity for fibrinogen, factor (F) X, FXIII, and combined FV and FVIII deficiencies. A weaker association was present for FV and FVII deficiencies. There was no association between coagulation factor activity level and clinical bleeding severity for FXI. The coagulation factor activity levels that were necessary for patients to remain asymptomatic were: fibrinogen, > 100 mg dL(-1); FV, 12 U dL(-1); combined FV + VIII, 43 U dL(-1); FVII, 25 U dL(-1); FX, 56 U dL(-1); FXI, 26 U dL(-1); FXIII, 31 U dL(-1). Moreover, coagulation factor activity levels that corresponded with Grade III bleeding were: undetectable levels for fibrinogen, FV and FXIII, < 15 U dL(-1) for combined FV + VIII; < 8 U dL(-1) for FVI; < 10 U dL(-1) for FX; and < 25 U dL(-1) for FXI. Conclusions: There is a heterogeneous association between coagulation factor activity level and clinical bleeding severity in different RBDs. A strong association is only observed in fibrinogen, FX and FXIII deficiencies.