The Von Willebrand Disease (VWD) International Prophylaxis Study (VIP)

Skane University Hospital

Status: Completed

Conditions: Von Willebrand Disease

Treatments: Drug: VWF/FVIII products

Study type: Observational

Funder types: Other; Industry

Identifiers

NCT00557908

VWD PN

Details and patient eligibility

About

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).

Full description

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.

Enrollment: 105 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

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 1. Three or more bleeding episodes in a six-month period that required treatment with VWD concentrates or red cell transfusions.

Trial design

105 participants in 1 patient group

VWF/FVIII product infusions

Description:

One to three infusions of factor replacement as needed to control bleeding.

Treatment:

Drug: VWF/FVIII products