The VWD International Prophylaxis (VIP) Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 105
- 试验地点
- 3
- 主要终点
- von Willebrand Disease associated bleeding frequency
研究概览
简要总结
The von Willebrand Disease Prophylaxis Network (VWD PN) is an international study group formed with the goal of investigating the role of prophylaxis in clinically severe VWD that is non-responsive to other treatment(s).
详细描述
The most common indications for vWD prophylaxis included joint bleeding, epistaxis, gastrointestinal (GI) bleeding, and menorrhagia. Thus, an effort to establish optimal treatment regimens for these indications, through a period of prospective evaluation, is the primary focus of this research. Other goals include a retrospective study of the effect of prophylaxis on bleeding frequency, and a retrospective natural history study of GI bleeding in VWD.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Type 1: eligible for participation if
- •≤20% RCo and/or ≤20% FVIII; and
- •DDAVP non-responsive, defined as occurrence of bleeding episodes not responding satisfactorily to desmopressin, or deemed non-responsive a priori by the investigator; and
- •Bleeding indication criteria are met
- •Type 2: eligible for participation if
- •DDAVP non-responsive, defined as occurrence of bleeding episodes not responding satisfactorily to desmopressin, or deemed non-responsive a priori by the investigator; or Type 2B;
- •Bleeding indication criteria are met
- •Type 3: eligible for participation if
- •Bleeding indication criteria are met
- •Bleeding Indication Criteria:
- •Joint Bleeding: documentation of at least two apparently spontaneous bleeding episodes in the same joint in the six months prior to enrollment; or three or more apparently spontaneous bleeding episodes in different joints in the six months prior to enrollment.
- •GI Bleeding: history of two or more severe GI bleeding episodes associated with either a drop in hemoglobin of ≥ 2 g/dl or requiring red blood cell transfusion or treatment with VWD concentrate.
- •Failure to identify other causes of bleeding.
- •Menorrhagia: a diagnosis of menorrhagia; prospectively completed Pictorial Blood Assessment Chart score >185 or required treatment with a VWD product for menstrual bleeding on one or more occasions in the year prior to enrollment.
- •Normal cervical cytology (PAP) within the six months prior to enrollment for females ≥ 18 years of age.
- •Epistaxis
- •Three or more bleeding episodes in a six-month period that required treatment with VWD concentrates or red cell transfusions.
排除标准
- 未提供
研究组 & 干预措施
VWF/FVIII product infusions
One to three infusions of factor replacement as needed to control bleeding.
干预措施: VWF/FVIII products (Drug)
结局指标
主要结局
von Willebrand Disease associated bleeding frequency
时间窗: 1 year
次要结局
- Optimal treatment regimens for joint bleeding, GI bleeding, epistaxis, and menorrhagia(1 year)
研究者
Sharyne M. Donfield, Ph.D.
Principal Research Scientist
Skane University Hospital