Summary
In patients with von Willebrand disease (VWD) replacement therapy with factor VIII/von
Willebrand (VWF) concentrates is increasingly applied as prophylactic regimen. Since
2000, 82 consecutively enrolled patients with clinically relevant bleeding episodes
(spontaneous, peri- or postoperative) were diagnosed with VWD [type 1: 42/82; type
2: 24/82; type 3: 13/82; acquired: 3/82]. In all patients, decision for initiating
prophylaxis was based on a bleeding score > 2 prior to diagnosis, concomitant with
recurrent bleeds associated with anaemia in patients with on-demand VWD therapy. We
report results on secondary prophylactic VWF replacement therapy applied in 32 patients
[children n=13; adolescents n=7; adults n=12] with VWD [type 1: 4; type 2: 15; type
3: 13], 15 of which were females, and nine of these at the reproductive period. Eight
patients were treated with Humate P→ or Wilate→ (n=24). Median [min-max] dose [vWF:RCo] was 40 [20–47] IU/kg, 23 patients were given
substitution therapy twice weekly, seven patients three times a week, and two children
four times per week. Within a 12-month-period haemoglobin concentrations returned
to normal values. Median duration of prophylaxis was three years. Recurrent bleeding
episodes stopped in 31 of 32 patients, whereas inhibitors developed in one. Following
a 12-month observation period the monthly bleeding frequency and the bleeding score
was significantly reduced [3 vs. 0.07; 3 vs. 0: p< 0.001], compared to the preprophylaxis/pre-diagnostic
values. The use of secondary prophylactic VWF replacement therapy is an effective
tolerated treatment modality, highly beneficial for patients with VWD, who present
with recurrent bleeding events during on-demand therapy.
Keywords
von Willebrand Disease - children - young adults - long-term prophylaxis - inhibitor
development
