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)

Vonicog Alfa (Recombinant Von Willebrand Factor) Treatment Outcomes in Von Willebrand Disease in the UK: a Retrospective Chart Review Study

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.).

Study Overview

• Status: Completed
• Conditions: Von Willebrand Disease (VWD)
• Intervention / Treatment: Other: No Intervention

• Study Type: Observational
• Enrollment (Actual): 34

Contacts and Locations

Study Locations

• United Kingdom
  • Birmingham, United Kingdom, B15 2GW
    • University Hospitals Birmingham
  • Leeds, United Kingdom, LS9 7TF
    • Leeds Teaching Hospital
  • Liverpool, United Kingdom, L7 8XP
    • Liverpool University Hospital
  • London, United Kingdom, NW3 2QG
    • Royal Free London
  • London, United Kingdom, W2 1NY
    • Imperial College Healthcare
  • Manchester, United Kingdom, M13 9WL
    • Manchester University
  • Oxford, United Kingdom, OX3 9DU
    • Oxford University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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; This is a non-interventional study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With VWD-Related Healthcare Resource Utilization (HRU)	HRU will include bleeding-related hospitalization rates, outpatient visits, accident and emergency visits.	Up to 24 months
Number of Participants With Surgery Related Costs By Type	Surgery related costs by type will include length of stay in intensive care unit (ICU), rVWF consumption, factor VIII (FVIII) consumption, VWD treatment consumption, laboratory tests and examinations.	Up to 24 months
Number of Participants With Bleeding Episodes Treated With rVWF	Bleeding episodes will be reported based on type, severity, location, treatment, and outcomes, of all recorded bleeds within 12 months prior to and 12 months following the first administration of rVWF.	Up to 24 months
Number of Surgical Procedures	Surgical procedures will be reported based on surgery type (orthopaedic, gastro-intestinal, dental, etc), severity (major, minor), category (emergency or elective) treatment duration and outcomes, of all recorded surgeries within 12 months prior to and 12 months following the first administration of rVWF.	Up to 24 months
Number of Participants with Surgery Outcomes (Success, Failure, Complications)	Surgery outcomes (success, failure, complications) based on average number of post-operative bleeds will be reported.	Up to 24 months

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Study Director, Takeda

Study record dates

Study Major Dates

• Study Start (Actual): December 16, 2021
• Primary Completion (Actual): October 30, 2023
• Study Completion (Actual): October 30, 2023

Study Registration Dates

• First Submitted: May 23, 2024
• First Submitted That Met QC Criteria: May 23, 2024
• First Posted (Actual): May 30, 2024

Study Record Updates

• Last Update Posted (Actual): May 30, 2024
• Last Update Submitted That Met QC Criteria: May 23, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Keywords: Drug Therapy
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Blood Platelet Disorders; Von Willebrand Diseases
• Other Study ID Numbers: TAK-577-5001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
• IPD Sharing Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No