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: Male; Factor VIII; Adolescent; Hemorrhage; 3. Good health; Cohort Studies; Hemoglobins; Young Adult; von Willebrand Diseases; 03 medical and health sciences; Treatment Outcome; 0302 clinical medicine; Clinical Protocols; Humans; Female; Child; Follow-Up Studies

SummaryIn 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.