Title :

Prophylaxis with recombinant von Willebrand factor in patients with type 3 von Willebrand disease: Results of a post hoc analysis from a phase 3 trial

Author(s) :

Leebeek, Frank [Auteur]
Peyvandi, Flora [Auteur]
Tiede, Andreas [Auteur]
Medizinische Hochschule Hannover = Hannover Medical School [MHH]
Castaman, Giancarlo [Auteur]
Escobar, Miguel [Auteur]
Wang, Michael [Auteur]
The University of Texas M.D. Anderson Cancer Center [Houston]
Zülfikar, Bulent [Auteur]
Istanbul University
Susen, Sophie [Auteur]
Récepteurs Nucléaires, Maladies Métaboliques et Cardiovasculaires - U 1011 [RNMCD]
Miesbach, Wolfgang [Auteur]
Wang, Scarlett [Auteur]
Wang, Yi [Auteur]
Zhang, Jingmei [Auteur]
Özen, Gülden [Auteur]

Journal title :

European Journal of Haematology

Pages :

29-40

Publisher :

Wiley

Publication date :

2023-04-11

ISSN :

0902-4441

HAL domain(s) :

Sciences du Vivant [q-bio]/Médecine humaine et pathologie/Cardiologie et système cardiovasculaire

English abstract : [en]

Abstract:Objectives To describe efficacy/safety of recombinant von Willebrand factor (rVWF) prophylaxis in patients with type 3 von Willebrand disease (VWD).Methods:This post hoc analysis of a phase 3 open‐label trial ...
Show more >Abstract:Objectives To describe efficacy/safety of recombinant von Willebrand factor (rVWF) prophylaxis in patients with type 3 von Willebrand disease (VWD).Methods:This post hoc analysis of a phase 3 open‐label trial provides a more detailed analysis of adults with type 3 VWD, categorized based on prior treatment at screening: “Prior On‐Demand (OD)” (OD VWF; ≥3 documented spontaneous bleeding events [BEs] requiring VWF in previous 12 months) or “Switch” (plasma‐derived [pd] VWF prophylaxis for ≥12 months). Annualized bleeding rates (ABRs) were evaluated during 12 months of rVWF prophylaxis versus historical data from medical records.Results:In the Prior OD group ( n = 10), mean spontaneous ABR (sABR) for treated BEs was reduced by 91.6% (ratio, 0.08; 95% CI, 0.02–0.45) versus mean historical sABR. In the Switch group ( n = 8), mean sABR for treated BEs was reduced by 47% (ratio, 0.53; 95% CI, 0.08–3.62). One non‐serious adverse event (AE) was considered possibly related to rVWF. No treatment‐related, fatal, or life‐threatening serious AEs were reported, and no patient developed VWF inhibitors.Conclusions:rVWF prophylaxis reduced sABR in type 3 VWD patients previously treated with OD VWF therapy, and maintained a similar level of hemostatic control in those switching from pdVWF prophylaxis to rVWF prophylaxis.Show less >

Language :

Anglais

Peer reviewed article :

Oui

Audience :

Internationale

Popular science :

Non

Collections :

• Récepteurs nucléaires, Maladies Cardiovasculaires et Diabète (RNMCD) - U1011

Source :

Harvested from HAL