Department of Haematology, Erasmus University Medical Centre, Rotterdam, The Netherlands.
Department of Paediatric Haematology, Erasmus University Medical Centre-Sophia Children's Hospital, Rotterdam, The Netherlands.
Haemophilia. 2020 Sep;26(5):916-922. doi: 10.1111/hae.14106. Epub 2020 Jul 26.
Diagnosis, treatment monitoring and assessment of desmopressin effect in haemophilia A patients are performed by measurement of factor VIII activity (FVIII). The two assays commonly applied are the one-stage assay and the chromogenic assay. Especially in non-severe haemophilia A, discrepancies between these assays are common. It is still unestablished which assay corresponds best with bleeding phenotype and desmopressin effect.
To correlate FVIII levels measured by the one-stage assay and by the chromogenic assay with bleeding phenotype and, additionally, to compare FVIII assay discrepancies before and after desmopressin administration.
Factor VIII was measured in 130 non-severe haemophilia A patients during routine visits to the outpatient clinic and/or during desmopressin testing. FVIII was measured by both the one-stage assay and the chromogenic assay. Discrepancies between assays were defined as at least a twofold difference of FVIII or an absolute FVIII difference between measurements of ≥0.10 IU/mL. Bleeding phenotype was defined as annual number of treated bleedings (adjusted ABR).
Hundred and thirty non-severe haemophilia A patients were included. In 31/130 patients, assay results were discrepant. However, FVIII measurements with both assays correlated adequately with adjusted ABR. In addition, in 27/130 patients FVIII measurements at baseline and after desmopressin administration were analysed. In 13/27 patients, all measurements were either equivalent or discrepant when results were compared. In 14/27 patients, this was not the case as both equivalent measurements and discrepant measurements at different time points within one patient were observed.
Neither the one-stage assay nor the chromogenic assay is superior in predicting bleeding phenotype. In addition, equivalent or discrepant FVIII results measured before desmopressin do not always predict FVIII assay results after desmopressin administration.
对血友病 A 患者的去氨加压素效果进行诊断、治疗监测和评估是通过测量因子 VIII 活性（FVIII）来进行的。常用的两种检测方法是一步法检测和显色法检测。特别是在非重度血友病 A 中，这两种检测方法之间的差异很常见。哪种检测方法与出血表型和去氨加压素效果最相关仍然没有定论。
通过一步法检测和显色法检测来比较 FVIII 水平与出血表型的相关性，并比较去氨加压素治疗前后 FVIII 检测方法的差异。
在非重度血友病 A 患者的常规门诊就诊期间和/或去氨加压素检测期间，我们测量了 130 名非重度血友病 A 患者的 FVIII。FVIII 采用一步法和显色法进行检测。将检测方法之间的差异定义为 FVIII 至少相差两倍或两次测量的 FVIII 绝对差值≥0.10 IU/mL。出血表型定义为每年治疗出血的次数（校正 ABR）。
共纳入 130 名非重度血友病 A 患者。在 130 名患者中，有 31 名患者的检测结果存在差异。然而，两种检测方法的 FVIII 测量结果与校正 ABR 相关性良好。此外，在 130 名患者中有 27 名患者的基线和去氨加压素治疗后的 FVIII 测量值进行了分析。在 27 名患者中，有 13 名患者的所有测量值要么等效，要么在不同时间点存在差异。在 27 名患者中有 14 名患者的情况并非如此，因为同一患者的不同时间点既存在等效测量值，也存在差异测量值。
一步法检测和显色法检测在预测出血表型方面都没有优势。此外，去氨加压素治疗前后等效或差异的 FVIII 结果并不总是预测去氨加压素治疗后的 FVIII 检测结果。
