Goldberg Nicola, Nisenbaum Rosane, Song Hong, Lillicrap David, Teitel Jerome, James Paula, Sholzberg Michelle
Department of Medicine University of Toronto Toronto ON Canada.
Li Ka Shing Knowledge Institute St Michael's Hospital Toronto ON Canada.
Res Pract Thromb Haemost. 2020 May 30;4(6):1046-1052. doi: 10.1002/rth2.12354. eCollection 2020 Aug.
Patients with type 1 von Willebrand disease (VWD) undergo a desmopressin (DDAVP) responsiveness challenge at diagnosis to assess whether DDAVP reverses their coagulation deficits. Current practice assumes DDAVP responsiveness remains constant over the lifetime. In patients with type 1 VWD, VWF-related parameters increase with age. This study explores whether DDAVP responsiveness also differs with age in this population.
We conducted a retrospective chart review of 106 patients enrolled at our center since 1990. Our primary outcome was DDAVP responsiveness at 1 hour after DDAVP challenge. Locally weighted scatterplot smoothing fit and Spearman correlation coefficients were used to study the relationship between age and DDAVP responsiveness. For female participants, we used the Kruskal-Wallis test to compare absolute and relative changes in DDAVP responsiveness at various ages.
We had 79 patients (56 female) with type 1 VWD with at least 1 DDAVP challenge. In women with type 1 VWD, the absolute change in DDAVP responsiveness did not vary significantly with age (VWF:antigen [Ag], -0.08, = .56; VWF:ristocetin cofactor [RCo], -0.16, = .26; low-molecular-weight component of factor VIII [FVIII:C], -0.01, = .93), nor did the relative change in DDAVP responsiveness (VWF:Ag, -0.03, = .86; VWF:RCo, -0.25, = .09; FVIII:C, -0.14, = .34). The data plot suggested a relationship.
In women with type 1 VWD, DDAVP responsiveness may vary over the life cycle. Our exploratory findings are limited by our retrospective data, cross-sectional design, and small sample. Future studies should investigate the relationship between age and DDAVP responsiveness prospectively to evaluate whether there is clinical utility in rechallenging postpubertal female patients with type 1 VWD.
1型血管性血友病（VWD）患者在诊断时需接受去氨加压素（DDAVP）反应性挑战，以评估DDAVP是否能逆转其凝血缺陷。目前的做法是假定DDAVP反应性在患者一生中保持不变。在1型VWD患者中，血管性血友病因子（VWF）相关参数会随年龄增长而增加。本研究探讨在该人群中DDAVP反应性是否也会随年龄而不同。
我们对自1990年起在本中心登记的106例患者进行了回顾性病历审查。我们的主要结局是DDAVP激发后1小时的DDAVP反应性。使用局部加权散点图平滑拟合和Spearman相关系数来研究年龄与DDAVP反应性之间的关系。对于女性参与者，我们使用Kruskal-Wallis检验来比较不同年龄时DDAVP反应性的绝对变化和相对变化。
我们纳入了79例至少接受过1次DDAVP激发的1型VWD患者（56例女性）。在1型VWD女性患者中，DDAVP反应性的绝对变化随年龄无显著差异（VWF：抗原[Ag]，-0.08，P = 0.56；VWF：瑞斯托霉素辅因子[RCo]，-0.16，P = 0.26；因子VIII低分子量成分[FVIII：C]，-0.01，P = 0.93），DDAVP反应性的相对变化也无显著差异（VWF：Ag，-0.03，P = 0.86；VWF：RCo，-0.25，P = 0.09；FVIII：C，-0.14，P = 0.34）。数据图显示存在某种关系。
在1型VWD女性患者中，DDAVP反应性可能在生命周期中有所不同。我们的探索性研究结果受回顾性数据、横断面设计和小样本的限制。未来的研究应前瞻性地调查年龄与DDAVP反应性之间的关系，以评估对青春期后1型VWD女性患者再次进行激发试验是否具有临床实用性。
