当前位置： SCI文献检索 > THROMBOSIS AND HAEMOSTASIS期刊下所有文献 > Long-term secondary prophylaxis in children, adolescents and young adults with von Willebrand disease. Results of a cohort study.

Long-term secondary prophylaxis in children, adolescents and young adults with von Willebrand disease. Results of a cohort study.

Abstract：

: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 pre-prophylaxis/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.

journal_name

Thromb Haemost

journal_title

Thrombosis and haemostasis

authors

Halimeh S,Krümpel A,Rott H,Bogdanova N,Budde U,Manner D,Faeser B,Mesters R,Nowak-Göttl U

doi

10.1160/TH10-09-0616

subject

Has Abstract

pub_date

2011-04-01 00:00:00

pages

597-604

issue

eissn

0340-6245

issn

2567-689X

pii

10-09-0616

journal_volume

105

pub_type

杂志文章

在线工具

Long-term secondary prophylaxis in children, adolescents and young adults with von Willebrand disease. Results of a cohort study.