prikaz prve stranice dokumenta von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease
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Scientific paper - Original scientific paper
von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease
Blood Advances, 8 (2024), 6; 1405-1414. https://doi.org/10.1182/bloodadvances.2023011742
Sidonio, Robert F.; Boban, Ana; Dubey, Leonid; Inati, Adlette; Kiss, Csongor; Boda, Zoltan; Lissitchkov, Toshko; Nemes, Laszlo; Novik, Dzmitry; Peteva, Elina; Taher, Ali T.; Timofeeva, Margarita Arkadevna; Vilchevska, Kateryna V.; Vdovin, Vladimir; Werner, Sylvia; Knaub, Sigurd; Djambas Khayat, Claudia More authors...

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Sidonio, R. F., Boban, A., Dubey, L., Inati, A., Kiss, C., Boda, Z. ... Djambas Khayat, C. (2024). von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease. Blood Advances, 8. (6), 1405-1414. doi: 10.1182/bloodadvances.2023011742

Sidonio, Robert F., et al. "von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease." Blood Advances, vol. 8, no. 6, 2024, pp. 1405-1414. https://doi.org/10.1182/bloodadvances.2023011742

Sidonio, Robert F., Ana Boban, Leonid Dubey, Adlette Inati, Csongor Kiss, Zoltan Boda, Toshko Lissitchkov, et al. "von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease." Blood Advances 8, no. 6 (2024): 1405-1414. https://doi.org/10.1182/bloodadvances.2023011742

Sidonio, R. F., et al. (2024) 'von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease', Blood Advances, 8(6), pp. 1405-1414. doi: 10.1182/bloodadvances.2023011742

Sidonio RF, Boban A, Dubey L, Inati A, Kiss C, Boda Z, and sur.. von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease. Blood Advances [Internet]. 2024 March 26 [cited 2024 December 30];8(6):1405-1414. doi: 10.1182/bloodadvances.2023011742

R. F. Sidonio, et al., "von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease", Blood Advances, vol. 8, no. 6, pp. 1405-1414, March 2024. [Online]. Available at: https://urn.nsk.hr/urn:nbn:hr:105:801633. [Accessed: 30 December 2024]

Metadata
Title (english)von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease
AuthorRobert F. Sidonio
AuthorAna Boban
AuthorLeonid Dubey
AuthorAdlette Inati
AuthorCsongor Kiss
AuthorZoltan Boda
AuthorToshko Lissitchkov
AuthorLaszlo Nemes
AuthorDzmitry Novik
AuthorElina Peteva
AuthorAli T. Taher
AuthorMargarita Arkadevna Timofeeva
AuthorKateryna V. Vilchevska
AuthorVladimir Vdovin
AuthorSylvia Werner
AuthorSigurd Knaub
AuthorClaudia Djambas Khayat
Author's institutionUniversity of Zagreb
School of Medicine
(Department of Internal Medicine)
Scientific / art field,
discipline and subdiscipline		BIOMEDICINE AND HEALTHCARE
Clinical Medical Sciences
Internal Medicine
Abstract (english)
Long-term prophylaxis with a von Willebrand factor (VWF) concentrate is recommended in patients with von Willebrand disease (VWD) who have a history of severe and frequent bleeds. However, data from prospective studies are scarce. WIL-31, a prospective, noncontrolled, international phase 3 trial, investigated the efficacy and safety of Wilate prophylaxis in severe patients with VWD. Male and female patients 6 years or older with VWD types 1, 2 (except 2N), or 3 who had completed a prospective, 6-month, on-demand, run-in study (WIL-29) were eligible to receive Wilate prophylaxis for 12 months. At baseline, patients (n = 33) had a median age of 18 years. Six (18%) patients had severe type 1, 5 (15%) had type 2, and 22 (67%) had type 3 VWD. The primary end point of a >50% reduction in mean total annualized bleeding rate (TABR) with Wilate prophylaxis vs prior on-demand treatment was met; mean TABR during prophylaxis was 5.2, representing an 84.4% reduction. The bleeding reduction was consistent across age, sex, and VWD types. The mean spontaneous ABR was 3.2, representing an 86.9% reduction vs on-demand treatment. During prophylaxis, 10 (30.3%) patients had 0 bleeding events and 15 (45.5%) patients had 0 spontaneous bleeding events. Of 173 BEs, 84.4% were minor and 69.9% treated. No serious adverse events related to study treatment and no thrombotic events were recorded. Overall, WIL-31 showed that Wilate prophylaxis was efficacious and well-tolerated in pediatric and adult patients with VWD of all types. The WIL-29 and WIL-31 trials were registered at www.ClinicalTrials.gov as #NCT04053699 and #NCT04052698, respectively.
Keywords (english)
Languageenglish
Publication typeScientific paper - Original scientific paper
Publication statusPublished
Peer reviewPeer review
Publication versionPublished version
Journal titleBlood Advances
Numberingvol. 8, no. 6, pp. 1405-1414
p-ISSN2473-9529
e-ISSN2473-9537
DOIhttps://doi.org/10.1182/bloodadvances.2023011742
URN:NBNurn:nbn:hr:105:801633
Publication2024-03-26
Document URLhttps://ashpublications.org/bloodadvances/article/8/6/1405/514639/von-Willebrand-factor-factor-VIII-concentrate
Type of resourceText
Access conditionsOpen access
Terms of useLicence Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0) icon
Created on2024-12-11 11:10:18