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)

NCT06433778 | Completed

• 1 other identifier: TAK-577-5001
• Study Type: observational
• Target: 34
• Locations: 1 country
• Sites: 7

Brief Summary

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.).

Conditions

Von Willebrand Disease (VWD)

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (3)

• Number of Participants With Bleeding Episodes Treated with rVWF Between the Index Date and Following 12 Months

  Bleeding episodes will be assessed based on categories of overall and annualized, type, severity, location, bleed frequency, bleed type/location and bleed severity.

  From index date up to 12 months

• Number of Surgical Procedures Between the Index Date and Following 12 Months

  Surgical procedures included surgery type, severity, category (emergency or elective). Data on the participants use of rVWF in the pre-, intra- and post-operative setting during the period between the index date and the following 12 months, will be stratified by VWD type, surgery type, severity, location, or treatment rationale.

  From index date up to 12 months

• Number of Participants with Surgery Outcomes (Success, Failure, Complications) Between the Index Date and Following 12 Months

  Surgery outcomes (success, failure, complications) will be reported.

  From index date up to 12 months

Secondary Outcomes (5)

• Number of Participants With VWD-Related Healthcare Resource Utilization (HRU)

  Up to 24 months

• Number of Participants With Surgery Related Costs By Type

  Up to 24 months

• Number of Participants With Bleeding Episodes Treated With rVWF

  Up to 24 months

• Number of Surgical Procedures

  Up to 24 months

• Number of Participants with Surgery Outcomes (Success, Failure, Complications)

  Up to 24 months

Study Arms (1)

Participants diagnosed With Congenital VWD

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.

Other: No Intervention

Interventions

No Intervention OTHER

This is a non-interventional study.

Participants diagnosed With Congenital VWD

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Participants who have been diagnosed with congenital VWD and were prescribed rVWF in UK.

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Takeda: lead

Study Sites (7)

University Hospitals Birmingham

Birmingham, B15 2GW, United Kingdom

Location

Leeds Teaching Hospital

Leeds, LS9 7TF, United Kingdom

Location

Liverpool University Hospital

Liverpool, L7 8XP, United Kingdom

Location

Royal Free London

London, NW3 2QG, United Kingdom

Location

Imperial College Healthcare

London, W2 1NY, United Kingdom

Location

Manchester University

Manchester, M13 9WL, United Kingdom

Location

Oxford University Hospital

Oxford, OX3 9DU, United Kingdom

Location

Related Links

• To obtain more information on the study, click here/on this link.

MeSH Terms

Conditions

von Willebrand Diseases

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 23, 2024
• First Posted: May 30, 2024
• Study Start: December 16, 2021
• Primary Completion: October 30, 2023
• Study Completion: October 30, 2023
• Last Updated: March 11, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

GB (7)