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)

Completed

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.).

Recruitment & Eligibility

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arm && Interventions

Group	Intervention	Description
Participants diagnosed With Congenital VWD	No Intervention	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.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.
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 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.

Locations (7): Leeds Teaching Hospital
🇬🇧
Leeds, United Kingdom
Manchester University
🇬🇧
Manchester, United Kingdom
Imperial College Healthcare
🇬🇧
London, United Kingdom
Liverpool University Hospital
🇬🇧
Liverpool, United Kingdom
Royal Free London
🇬🇧
London, United Kingdom
Oxford University Hospital
🇬🇧
Oxford, United Kingdom
University Hospitals Birmingham
🇬🇧
Birmingham, United Kingdom