PDF(Pubmed)
Abstract：
BACKGROUND: Hemophilia A (HA) is an X-linked inherited bleeding disorder caused by reduced factor VIII (FVIII) levels. Approximately 10-15% of patients with severe HA (SHA) do not present with the anticipated bleeding pattern. Here, we assessed the phenotypic severity of hemophilia A using rotational thromboelastometry (ROTEM) and activated partial thromboplastin time-clot waveform analysis (APTT-CWA).
METHODS: Patients diagnosed with hemophilia A were enrolled. Clinical phenotype assignment was performed according to the published literature, and patients were classified into four phenotypic subgroups. The whole blood sample was first run on ROTEM in INTEM mode using platelet-poor plasma, APTT was run, and the APTT-CWA graph was simultaneously recorded.
RESULTS: A total of 66 patients were recruited for this study. Statistically significant differences were observed between the four phenotypically categorized groups using ROTEM and APTT-CWA. On comparing patients with mild/moderate-to-severe phenotypes (Group II) with SHA without inhibitors (Group IV), no significant difference was found for all parameters of ROTEM or APTT-CWA. The MCF, MA30, MAXV, and Alpha angle values using ROTEM were found to be the lowest in patients with SHA with inhibitors, which helped differentiate them from those with SHA without inhibitors. However, these two groups could not be differentiated using the APTT-CWA parameters.
CONCLUSIONS: ROTEM can be used to distinguish patients with SHA with inhibitors from those with SHA without inhibitors using a combination of parameters with high sensitivity and specificity. However, APTT-CWA cannot be used to differentiate these patient groups.
METHODS: Patients diagnosed with hemophilia A were enrolled. Clinical phenotype assignment was performed according to the published literature, and patients were classified into four phenotypic subgroups. The whole blood sample was first run on ROTEM in INTEM mode using platelet-poor plasma, APTT was run, and the APTT-CWA graph was simultaneously recorded.
RESULTS: A total of 66 patients were recruited for this study. Statistically significant differences were observed between the four phenotypically categorized groups using ROTEM and APTT-CWA. On comparing patients with mild/moderate-to-severe phenotypes (Group II) with SHA without inhibitors (Group IV), no significant difference was found for all parameters of ROTEM or APTT-CWA. The MCF, MA30, MAXV, and Alpha angle values using ROTEM were found to be the lowest in patients with SHA with inhibitors, which helped differentiate them from those with SHA without inhibitors. However, these two groups could not be differentiated using the APTT-CWA parameters.
CONCLUSIONS: ROTEM can be used to distinguish patients with SHA with inhibitors from those with SHA without inhibitors using a combination of parameters with high sensitivity and specificity. However, APTT-CWA cannot be used to differentiate these patient groups.
摘要：
背景：血友病A（HA）是一种X连锁遗传性出血性疾病，由因子VIII（FVIII）水平降低引起。大约10-15％的重度HA（SHA）患者没有出现预期的出血模式。这里,我们使用旋转血栓弹性测定法(ROTEM)和活化部分凝血活酶时间-血凝块波形分析(APTT-CWA)评估了血友病A的表型严重程度.
方法：纳入诊断为A型血友病的患者。根据已发表的文献进行临床表型分配，将患者分为四个表型亚组。首先使用缺乏血小板的血浆在ROTEM上以INTEM模式运行全血样品，APTT运行了，同时记录APTT-CWA图。
结果：本研究共招募66名患者。使用ROTEM和APTT-CWA观察到四个表型分类组之间的统计学显著差异。在比较轻度/中度至重度表型（II组）与无抑制剂的SHA（IV组）的患者时，ROTEM或APTT-CWA的所有参数均未发现显着差异。MCF,MA30,MAXV,发现使用ROTEM的Alpha角度值在SHA患者中最低，这有助于将它们与没有抑制剂的SHA区分开。然而,使用APTT-CWA参数无法区分这两组.
结论：ROTEM可用于使用具有高灵敏度和特异性的参数组合来区分具有抑制剂的SHA患者与没有抑制剂的SHA患者。然而,APTT-CWA不能用于区分这些患者组。
方法：纳入诊断为A型血友病的患者。根据已发表的文献进行临床表型分配，将患者分为四个表型亚组。首先使用缺乏血小板的血浆在ROTEM上以INTEM模式运行全血样品，APTT运行了，同时记录APTT-CWA图。
结果：本研究共招募66名患者。使用ROTEM和APTT-CWA观察到四个表型分类组之间的统计学显著差异。在比较轻度/中度至重度表型（II组）与无抑制剂的SHA（IV组）的患者时，ROTEM或APTT-CWA的所有参数均未发现显着差异。MCF,MA30,MAXV,发现使用ROTEM的Alpha角度值在SHA患者中最低，这有助于将它们与没有抑制剂的SHA区分开。然而,使用APTT-CWA参数无法区分这两组.
结论：ROTEM可用于使用具有高灵敏度和特异性的参数组合来区分具有抑制剂的SHA患者与没有抑制剂的SHA患者。然而,APTT-CWA不能用于区分这些患者组。
