Long-term secondary prophylaxis in children, adolescents and young adults with von Willebrand disease

• DOI: 10.1160/th10-09-0616
• Publication Date: 2011-02-08T12:49:41Z
• Authors: Susan Halimeh; Anne Krümpel; Hannelore Rott; Nadja Bogdanova; Ulrich Budde; Daniela Manner; Britta Faeser; Rolf Mesters; Ulrike Nowak-Göttl
• Keywords: von Willebrand Disease; Regimen; Concomitant; Prophylactic treatment

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