A phase III study comparing secondary long-term prophylaxis versus on-demand treatment with vWF/FVIII concentrates in severe inherited von Willebrand disease.

ABSTRACT:

Background

There is a lack of prospective clinical trials specifically designed to evaluate the benefits of prophylaxis with vWF/FVIII concentrates in patients with inherited von Willebrand disease (vWD). The aim of the study was to compare efficacy of secondary long-term prophylaxis (PRO) with vWF/FVIII in the prevention of bleeding episodes in severe vWD patients to standard of care (on-demand treatment; ODT).

Materials and methods

In this 12-month, phase III, open-label study (PRO.WILL), vWD patients (aged ≥6 years) were randomised to PRO (n=9; 5 completed) or ODT (n=10; 7 completed) treatment with Fanhdi^®/Alphanate^® (Grifols) according to current licensing status for use in vWD. We assessed the proportion of patients who did not present any spontane

SUBMITTER: Peyvandi F 

PROVIDER: S-EPMC6774924 | biostudies-literature | 2019 Sep

REPOSITORIES: biostudies-literature