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Thromb Haemost 2011; 105(04): 597-604
DOI: 10.1160/TH10-09-0616
Blood Coagulation, Fibrinolysis and Cellular Haemostasis
Schattauer GmbH

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

Results of a cohort study
Susan Halimeh
1   Medical Thrombosis and Hemophilia treatment Center Duisburg, Germany
,
Anne Krümpel
2   Pediatric Hematology & Oncology, Univ. Hospital, University of Münster, Germany
,
Hannelore Rott
1   Medical Thrombosis and Hemophilia treatment Center Duisburg, Germany
,
Nadja Bogdanova
3   Institute of Medical Genetics, University of Münster, Germany
,
Ulrich Budde
4   Asklepios laboratory, Hospital Hamburg Altona, Hamburg, Germany
,
Daniela Manner
2   Pediatric Hematology & Oncology, Univ. Hospital, University of Münster, Germany
,
Britta Faeser
1   Medical Thrombosis and Hemophilia treatment Center Duisburg, Germany
,
Rolf Mesters
5   Department of Medicine, Hematology & Oncology, University Hospital, University of Münster, Germany
,
Ulrike Nowak-Göttl   *
2   Pediatric Hematology & Oncology, Univ. Hospital, University of Münster, Germany
› Author Affiliations
Further Information

Publication History

Received: 27 September 2010

Accepted after major revision: 10 February 2010

Publication Date:
28 November 2017 (online)

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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

* Present address: Head of the Coagulation & Hemostasis Unit, Institute of Clinical Chemistry, University Hospital Kiel & Lübeck, Kiel, Germany.


 

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