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. 2017 Nov;23(6):861-867. doi: 10.1111/hae.13295. Epub 2017 Jun 21.
Due to interindividual variation in desmopressin response, non-severe haemophilia A patients require desmopressin testing prior to therapeutic treatment. However, adequate response or frequency of blood sampling is not standardised in international guidelines. Consequently, various definitions and blood sampling protocols are currently applied. Interestingly, sustainability of desmopressin response is not incorporated into these definitions.
To study desmopressin response rates in a cohort of non-severe haemophilia A patients using currently accepted desmopressin response definitions. This, in order to formulate a standardised, uniform response which includes information on sustainability and to design a standardised blood sampling protocol.
Currently used desmopressin responses in non-severe haemophilia A patients were derived from a literature search. Actual desmopressin response rates were individualised in 105 non-severe HA patients from the Erasmus University Medical Centre and classified according to current varying definitions.
Five response definitions were evaluated, three of which included only factor VIII (FVIII):C cut-off levels and two also incorporated FVIII:C-fold increase over baseline.
C-fold increase showed no association with desmopressin response sustainability.
C 1 hour after infusion (<0.30, ≥0.30-0.49, ≥0.50-0.79 and ≥0.80 IU/mL) was, however, indicative of desmopressin response after 6 hours.
We suggest standardised desmopressin response based on clinically relevant FVIII:C levels, e.g. 0.30 and 0.50 IU/mL. In addition, patients with <0.30 IU/mL FVIII:C after 1 hour (non-responder) or ≥0.80 IU/mL (sustained responder) do not require subsequent blood sampling. However, patients with ≥0.30-0.79 IU/mL FVIII:C after 1 hour should undergo blood sampling after 6 hours to additionally determine response sustainability.
由于去氨加压素反应存在个体差异，非重度血友病 A 患者在治疗前需要进行去氨加压素测试。然而，国际指南中并未标准化充分反应或采血频率。因此，目前应用了各种定义和采血方案。有趣的是，这些定义中并未纳入去氨加压素反应的可持续性。
使用目前公认的去氨加压素反应定义，研究一组非重度血友病 A 患者的去氨加压素反应率。这是为了制定一个标准化、统一的反应定义，其中包括可持续性信息，并设计一个标准化的采血方案。
从文献检索中得出目前用于非重度血友病 A 患者的去氨加压素反应。从伊拉斯谟大学医学中心的 105 名非重度 HA 患者中个体化实际去氨加压素反应率，并根据当前不同的定义进行分类。
评估了五种反应定义，其中三种仅包括因子 VIII（FVIII）：C 截止水平，两种还包括 FVIII：C 相对于基线的增加倍数。FVIII：C 增加倍数与去氨加压素反应可持续性无关。
然而，输注后 1 小时 FVIII：C（<0.30、≥0.30-0.49、≥0.50-0.79 和≥0.80 IU/mL）可预测 6 小时后的去氨加压素反应。
我们建议基于临床相关 FVIII：C 水平（例如 0.30 和 0.50 IU/mL）标准化去氨加压素反应。此外，输注后 1 小时 FVIII：C <0.30 IU/mL（无反应者）或≥0.80 IU/mL（持续反应者）的患者无需进行后续采血。然而，输注后 1 小时 FVIII：C 为≥0.30-0.79 IU/mL 的患者应在 6 小时后进行采血，以进一步确定反应可持续性。
