Management of von Willebrand disease with a factor VIII-poor von Willebrand factor concentrate: Results from a prospective observational post-marketing study.

ABSTRACT:

Background

A triple-secured plasma-derived von Willebrand factor (pdVWF) almost devoid of factor VIII (FVIII):WILFACTIN^® , was approved in France in 2003, and then in other countries for the treatment of patients with von Willebrand disease (VWD).

Objective

To investigate long-term safety and efficacy of the product in real-life over the first 5 post-approval years.

Patients/methods

This prospective, observational, national post-marketing study (PMS) enrolled patients of all ages and VWD types. Patients were observed for up to 3 years and treated for one or more occasions. Efficacy was assessed for each major event. Breakthrough bleeding rate 3 days post-infusion and annualized bleeding rate (ABR) were also evaluated for long-term prophylaxis.

Results

SUBMITTER: Goudemand J 

PROVIDER: S-EPMC7496521 | biostudies-literature | 2020 Aug

REPOSITORIES: biostudies-literature