Von Willebrand Disease
Conditions
Keywords
Von Willebrand Disease, VWD, prophylaxis
Brief summary
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).
Detailed 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.
Interventions
DRUGVWF/FVIII products
Participants in the prospective phase of the study undergo an escalation of treatment from receipt of one to three levels of VWD product. All subjects enrolled will begin treatment on the level one and remain on this regimen for the duration of follow-up, or until they meet the criteria for escalation to level two or three (if indeed they do meet the criteria.) Dosing for joint bleeding, epistaxis, and GI bleeding indications: 50 U RCo/kg once per week, 50 U RCo/kg twice per week, or 50 U RCo/kg three times per week. Dosing for menorrhagia: 50 U RCo/kg on day 1 of menses for 2 cycles, 50 U RCo/kg on days 1 and 2 of menses for 2 cycles, or 50 U RCo/kg on days 1, 2, and 3 of menses
Sponsors
Versiti Blood Health
CSL Behring
Skane University Hospital
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| von Willebrand Disease associated bleeding frequency | 1 year |
Secondary
| Measure | Time frame |
|---|---|
| Optimal treatment regimens for joint bleeding, GI bleeding, epistaxis, and menorrhagia | 1 year |
Countries
Sweden, United States
Outcome results
None listed