Von Willebrand Disease (VWD)
Conditions
Keywords
Drug Therapy
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.).
Interventions
OTHERNo Intervention
This is a non-interventional study.
Sponsors
Takeda
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Surgical Procedures Between the Index Date and Following 12 Months | From index date up to 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. |
| Number of Participants With Bleeding Episodes Treated with rVWF Between the Index Date and Following 12 Months | From index date up to 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. |
| Number of Participants with Surgery Outcomes (Success, Failure, Complications) Between the Index Date and Following 12 Months | From index date up to 12 months | Surgery outcomes (success, failure, complications) will be reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Bleeding Episodes Treated With rVWF | Up to 24 months | 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. |
| Number of Surgical Procedures | Up to 24 months | 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. |
| Number of Participants with Surgery Outcomes (Success, Failure, Complications) | Up to 24 months | Surgery outcomes (success, failure, complications) based on average number of post-operative bleeds will be reported. |
| Number of Participants With VWD-Related Healthcare Resource Utilization (HRU) | Up to 24 months | HRU will include bleeding-related hospitalization rates, outpatient visits, accident and emergency visits. |
| Number of Participants With Surgery Related Costs By Type | Up to 24 months | 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. |
Countries
United Kingdom
Contacts
STUDY_DIRECTORStudy Director
Takeda
Outcome results
None listed