Factor VIII and von Willebrand activity are strongly related to the desmopressin biological response in von Willebrand disease

SÁNCHEZ-LUCEROS A; MESCHENGIESER SS; WOODS AI; GROSSO SH; LAZZARI MA

Boston-USA

Congreso; XXII Congress of the International Society on Thrombosis and Haemostasis; 2009

International Society of Thrombosis and Haemostasis

IntroductionDesmopressin is the first option to treat von Willebrand’s disease (VWD). Our aim was to collect data on the biological response to DDAVP to identify variables related to the DDAVP response, in a cohort of VWD patients from a single institution. MethodsA retrospective survey of data records (N= 783, 537 women, mean age 25.7, range 2-66 yrs) was performed to retrieve information on the biological response to intravenous DDAVP challenge test (0.3 mg kg-1 body weight). All patients were diagnosed according to the updated ISTH criteria; VWD 2N was excluded. Good response to DDAVP was defined as patients who, 1 and 2 hours after DDAVP infusion, had an increase of plasma FVIII:C and VWF:RCo of at least 3-fold over baseline and reached levels of at least 50 IU dL-1 for both values, with normal BT.Results746 patients had type 1 VWD (140 type 1 VWD, 606 Low VWF) and 37 type 2 VWD. Mean bleeding score was 2.98 ± 1.6; 87 patients had been transfused previous to diagnosis and 50 patients were asymptomatic. Overall 91.4% patients showed good response, being significantly different between groups: 81.4% in type 1, 95.5% in low VWF and 62.1% in type 2 VWD (p<0.001). Patients with FVIII:C and VWF:RCo lower than 30 IU dL-1 were at risk of DDAVP failure (RR 3.82, CI95:2.44-5.96; RR 5.02, CI95: 3.19-7.91; p<0.001). Children younger than 10 years showed non-response more frequently than older patients (16.9% vs. 4.9%; p<0.001). No patient showed significant unwanted effects.ConclusionsWe would define 3 markers related to the DDAVP responsiveness: age, FVIII and VWF:RCo levels. Children under 10 years appear to have higher probability of inadequate response. The age could reflect more than a DDAVP failure, a severity of the illness in this younger population. FVIII:C and VWF:RCo lower than 30IU dL-1 were related to inadequate response more frequently.