Vonicog Alfa (Recombinant Von Willebrand Factor) Treatment Outcomes in Von Willebrand Disease in the UK: a Retrospective Chart Review Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 34
- 试验地点
- 7
- 主要终点
- Number of Surgical Procedures Between the Index Date and Following 12 Months
研究概览
简要总结
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.).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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.
研究组 & 干预措施
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.
干预措施: No Intervention (Other)
结局指标
主要结局
Number of Surgical Procedures Between the Index Date and Following 12 Months
时间窗: 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
时间窗: 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
时间窗: From index date up to 12 months
Surgery outcomes (success, failure, complications) will be reported.
次要结局
- Number of Participants With Surgery Related Costs By Type(Up to 24 months)
- Number of Participants With VWD-Related Healthcare Resource Utilization (HRU)(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)