Desmopressin responsiveness by age in type 1 von Willebrand disease

Abstract

Background: 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.

Methods: 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.

Results: 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, P = .56; VWF:ristocetin cofactor [RCo], -0.16, P = .26; low-molecular-weight component of factor VIII [FVIII:C], -0.01, P = .93), nor did the relative change in DDAVP responsiveness (VWF:Ag, -0.03, P = .86; VWF:RCo, -0.25, P = .09; FVIII:C, -0.14, P = .34). The data plot suggested a relationship.

Conclusion: 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.

Keywords: deamino arginine vasopressin; hemorrhage; hemostasis; von Willebrand disease; women.