A Study on the Outcomes of Recombinant Von Willebrand Factor on Demand Treatment and Prevention and Treatment of Bleeding During and After Surgery in Adults With Inherited Von Willebrand Disease in the United Kingdom (UK)

Takeda

Status: Completed

Conditions: Von Willebrand Disease (VWD)

Treatments: Other: No Intervention

Study type: Observational

Funder types: Industry

Identifiers

NCT06433778

TAK-577-5001

Details and patient eligibility

About

This study is a retrospective chart review study and will collect data on real world use of vonicog alfa (Recombinant Von Willebrand Factor [rVWF]). Von Willebrand disease (VWD) is the most common inherited bleeding disorder. rVWF is approved in Europe and UK to treat bleeding and to treat and prevent bleeding during surgeries in adults in 2018.

This study will review and collect information on the treatment and bleed prevention of adult persons with inherited VWD with rVWF in UK. These data were already collected as a part of the routine care.

The main aims of this study are to describe the use of rVWF in on-demand treatment of bleeding and the prevention of treatment and treatment of bleeding during surgeries. Other aims are to describe bleedings and their treatment as well as any surgeries before and after first treatment with rVWF and to gather information on the use of healthcare resources (such as hospital visits, emergency room visits, etc.).

Enrollment: 34 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Adults (aged 18 or over at time of first administration of rVWF) who have provided informed consent and used rVWF within its licensed indication.

• Participants who have been diagnosed with congenital von Willebrand disease.

• Confirmed instance of

  • at least one bleed (either a new bleed or ongoing bleed treated under a treatment switch) treated on-demand with rVWF between 01-Oct-2020 and 30-Jun- 2022 and/or
  • treatment to prevent and treat surgical bleeds with rVWF between 01-Oct-2020 and 30-Jun-2022

Exclusion criteria

• Participants who were aged 17 years or less at the time of the first administration of rVWF.
• Participants who have been diagnosed with any other bleeding disorders or factor deficiencies including acquired von Willebrand disease.
• Participants with neutralising antibodies/inhibitors to VWF.
• Participants participation in a clinical trial of an investigational medical product during the study period.

Trial design

34 participants in 1 patient group

Participants diagnosed With Congenital VWD

Description:

Participants who have been diagnosed with congenital VWD and prescribed rVWF within the index date range (defined as the first administration of rVWF and must fall between 1st October 2020 and 30th June 2022) will be assessed using data obtained from medical records to evaluate the treatment outcome of rVWF in real-world clinical practice.

Treatment:

Other: No Intervention