The bleeding phenotype in people with nonsevere hemophilia.
The bleeding phenotype in people with nonsevere hemophilia.
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DOI：
10.1182/bloodadvances.2022007620
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发表时间：
2022-07-26
期刊：
影响因子：
7.5
通讯作者：
Gouw, Samantha C.
中科院分区：
文献类型：
--
作者：
Kloosterman, Fabienne R.;Zwagemaker, Anne-Fleur;Bagot, Catherine N.;Beckers, Erik A. M.;Castaman, Giancarlo;Cnossen, Marjon H.;Collins, Peter W.;Hay, Charles;Hof, Michel;Laros-van Gorkom, Britta;Leebeek, Frank W. G.;Male, Christoph;Meijer, Karina;Pabinger, Ingrid;Shapiro, Susan;Coppens, Michiel;Fijnvandraat, Karin;Gouw, Samantha C.
报告了非严重血友病患者出血率的估计值以及基线FVIII/IX与关节出血率之间的关系。一半的非严重血友病患者在过去经历过关节出血，尽管关节出血率很低。关于非严重血友病出血的发病、频率和严重程度的详细信息是有限的。我们的目的是评估非严重血友病患者的出血表型，并分析基线因子VIII/IX （FVIII/IX）水平与关节出血率之间的关系。在DYNAMO（中等和轻度血友病A和B出血表型与基线因子水平之间的动态相互作用）研究中，我们纳入了12至55岁的非严重血友病（FVIII/IX, 0.02-0.35 IU/mL）男性。从医疗档案中收集首次治疗（关节）出血的年龄、年出血率（ABRs）和年关节出血率（ajbr）的信息。基线FVIII/IX水平与关节出血率之间的关系通过使用复发事件的脆弱性模型进行评估。共纳入304人（70名中度血友病患者和234名轻度血友病患者）。中位年龄为38岁（四分位数范围[IQR]， 25-49岁），中位基线FVIII/IX水平为0.12 IU/mL （IQR, 0.05-0.21 IU/mL）。总共有245人（81%）经历过至少一次出血，156人（51%）经历过至少一次关节出血。首次出血和首次关节出血的中位年龄分别为8岁和10岁。中位ABR和AJBR分别为0.2 （IQR, 0.1-0.5）和0.0 （IQR, 0.0-0.2）。从基线FVIII/IX水平0.02 - 0.05 IU/mL降至bb0 - 0.25 IU/mL，中位ABR从0.6 （IQR, 0.2-1.4）降至0.1 (IQR, 0.0-0.2)， AJBR从0.2 （IQR, 0.0-0.4）降至0.0 （IQR, 0.0-0.0）。基线FVIII/IX与关节出血率呈负相关（P < 0.001）。在非严重血友病患者中观察到低出血率。然而，一半的青少年和成年人经历过关节出血。
Estimates for bleeding rates and the association between baseline FVIII/IX and joint bleeding rates in nonsevere hemophilia are reported. One-half of included persons with nonsevere hemophilia experienced a joint bleed in the past, despite low joint bleeding rates. Detailed information on the onset, frequency, and severity of bleeding in nonsevere hemophilia is limited. We aimed to assess the bleeding phenotype of persons with nonsevere hemophilia and to analyze the association between baseline factor VIII/IX (FVIII/IX) levels and the joint bleeding rate. In the DYNAMO (Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B) study, an international multicenter cohort, we included males with nonsevere hemophilia (FVIII/IX, 0.02-0.35 IU/mL) aged 12 to 55 years. Information on age at first treated (joint) bleed, annual bleeding rates (ABRs), and annual joint bleeding rates (AJBRs) was collected from the medical files. The association between baseline FVIII/IX levels and the joint bleeding rate was assessed by using a frailty model for recurrent events. In total, 304 persons (70 with moderate hemophilia and 234 with mild hemophilia) were included. The median age was 38 years (interquartile range [IQR], 25-49 years), and the median baseline FVIII/IX level was 0.12 IU/mL (IQR, 0.05-0.21 IU/mL). In total, 245 (81%) persons had experienced at least 1 bleed, and 156 (51%) had experienced at least 1 joint bleed. The median age at first bleed and first joint bleed was 8 and 10 years, respectively. The median ABR and AJBR was 0.2 (IQR, 0.1-0.5) and 0.0 (IQR, 0.0-0.2). From baseline FVIII/IX levels 0.02 to 0.05 IU/mL to >0.25 IU/mL, the median ABR decreased from 0.6 (IQR, 0.2-1.4) to 0.1 (IQR, 0.0-0.2) and the AJBR from 0.2 (IQR, 0.0-0.4) to 0.0 (IQR, 0.0-0.0). Baseline FVIII/IX was inversely associated with the joint bleeding rate (P < .001). Low bleeding rates were observed in persons with nonsevere hemophilia. However, one-half of all adolescents and adults had experienced a joint bleed.
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