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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 Više autora...

Institucijski repozitorij: Dr Med - digitalni repozitorij Medicinskog fakulteta Sveučilišta u Zagrebu

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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, br. 6, 2024, str. 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, br. 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), str. 1405-1414. doi: 10.1182/bloodadvances.2023011742

Sidonio RF, Boban A, Dubey L, Inati A, Kiss C, Boda Z, i sur.. von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease. Blood Advances [Internet]. 26.03.2024. [pristupljeno 29.12.2024.];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, br. 6, str. 1405-1414, Ožujak 2024. [Online]. Dostupno na: https://urn.nsk.hr/urn:nbn:hr:105:801633. [Citirano: 29.12.2024.]

Za citiranje koristite ovu mrežnu adresu: https://urn.nsk.hr/urn:nbn:hr:105:801633
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Naslov (engleski)von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease
AutorRobert F. Sidonio
AutorAna Boban
AutorLeonid Dubey
AutorAdlette Inati
AutorCsongor Kiss
AutorZoltan Boda
AutorToshko Lissitchkov
AutorLaszlo Nemes
AutorDzmitry Novik
AutorElina Peteva
AutorAli T. Taher
AutorMargarita Arkadevna Timofeeva
AutorKateryna V. Vilchevska
AutorVladimir Vdovin
AutorSylvia Werner
AutorSigurd Knaub
AutorClaudia Djambas Khayat
Autorova ustanovaSveučilište u Zagrebu
Medicinski fakultet
(KATEDRA ZA INTERNU MEDICINU)
Znanstveno / umjetničko
područje, polje i grana		BIOMEDICINA I ZDRAVSTVO
Kliničke medicinske znanosti
Interna medicina
Sažetak (engleski)
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.
Ključne riječi (engleski)
Jezikengleski
Vrsta publikacijeZnanstveni rad - Izvorni znanstveni rad
Status objaveObjavljen
Vrsta recenzijeRecenziran
Verzija publikacijeObjavljena verzija rada (izdavačev PDF)
Naslov časopisaBlood Advances
Brojčani podacivol. 8, br. 6, str. 1405-1414
p-ISSN2473-9529
e-ISSN2473-9537
DOIhttps://doi.org/10.1182/bloodadvances.2023011742
URN:NBNurn:nbn:hr:105:801633
Datum objave publikacije2024-03-26
URL dokumentahttps://ashpublications.org/bloodadvances/article/8/6/1405/514639/von-Willebrand-factor-factor-VIII-concentrate
Vrsta resursaTekst
Prava pristupaOtvoreni pristup
Uvjeti korištenjaIkona licence Imenovanje-Nekomercijalno-Bez prerada 4.0 međunarodna (CC BY-NC-ND 4.0)
Datum i vrijeme pohrane2024-12-11 11:10:18