title: 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.
authors.affiliation: Medical Thrombosis and Hemophilia Treatment Center Duisburg, Germany.
authors.forename: Susan
authors.initials: S
authors.lastname: Halimeh
authors.forename: Anne
authors.initials: A
authors.lastname: Krümpel
authors.forename: Hannelore
authors.initials: H
authors.lastname: Rott
authors.forename: Nadja
authors.initials: N
authors.lastname: Bogdanova
authors.forename: Ulrich
authors.initials: U
authors.lastname: Budde
authors.forename: Daniela
authors.initials: D
authors.lastname: Manner
authors.forename: Britta
authors.initials: B
authors.lastname: Faeser
authors.forename: Rolf
authors.initials: R
authors.lastname: Mesters
authors.forename: Ulrike
authors.initials: U
authors.lastname: Nowak-Göttl
mesh_terms: D000293:Adolescent; D002648:Child; D002985:Clinical Protocols; D015331:Cohort Studies; D005169:Factor VIII / Q000008:administration & dosage*; D005260:Female; D005500:Follow-Up Studies; D006454:Hemoglobins / Q000378:metabolism*; D006470:Hemorrhage / Q000517:prevention & control*; D006801:Humans; D008297:Male; D016896:Treatment Outcome; D055815:Young Adult; D014842:von Willebrand Diseases / Q000097:blood* / Q000517:prevention & control*
chemical_list: D006454:Hemoglobins; C078147:F8 protein, human; D005169:Factor VIII