Clinical trial NCT00557908: The Von Willebrand Disease (VWD) International Prophylaxis Study
Official title: The VWD International Prophylaxis (VIP) Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Von Willebrand Disease
Keywords: Von Willebrand Disease
VWD
prophylaxis
Lead sponsor: Skane University Hospital
Sponsor class: OTHER
Collaborators: name: Versiti
name: CSL Behring
First posted: 2007-11-14
Enrollment: 105
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The VWD population includes those with Type 1 if \</=20% RCo and/or \</=20% FVIII; and DDAVP non-responsive; Type 2 if DDAVP non-responsive, or Type 2B; and Type 3; who meet bleeding indication criteria having defined patterns of gastrointestinal bleeding, joint bleeding, epistaxis, or menorrhagia.

Individuals already on prophylaxis for VWD, for any indication, and individuals who were on a regimen of prophylaxis for at least six months that was discontinued because it was no longer required, or those with a history of GI bleeding due either to proven angiodysplasia or unexplained by other factors.

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.

Eligibility 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.

Primary outcomes:
measure: von Willebrand Disease associated bleeding frequency
timeFrame: 1 year
Secondary outcomes:
measure: Optimal treatment regimens for joint bleeding, GI bleeding, epistaxis, and menorrhagia
timeFrame: 1 year
Other outcomes:
Locations:
city: Chapel Hill
country: United States
facility: Rho, Inc.
state: North Carolina
city: Milwaukee
country: United States
facility: BloodCenter of Wisconsin
state: Wisconsin
city: Malmö
country: Sweden
facility: Skåne University Hospital
Study officials:
affiliation: Skåne University Hospital, Malmö, Sweden
name: Erik Berntorp, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Versiti
name: Thomas Abshire, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Holm E, Abshire TC, Bowen J, Alvarez MT, Bolton-Maggs P, Carcao M, Federici AB, Gill JC, Halimeh S, Kempton C, Key NS, Kouides P, Lail A, Landorph A, Leebeek F, Makris M, Mannucci P, Mauser-Bunschoten EP, Nugent D, Valentino LA, Winikoff R, Berntorp E. Changes in bleeding patterns in von Willebrand disease after institution of long-term replacement therapy: results from the von Willebrand Disease Prophylaxis Network. Blood Coagul Fibrinolysis. 2015 Jun;26(4):383-8. doi: 10.1097/MBC.0000000000000257.
citation: Makris M, Federici AB, Mannucci PM, Bolton-Maggs PHB, Yee TT, Abshire T, Berntorp E. The natural history of occult or angiodysplastic gastrointestinal bleeding in von Willebrand disease. Haemophilia. 2015 May;21(3):338-342. doi: 10.1111/hae.12571. Epub 2014 Nov 7.
citation: Abshire TC, Federici AB, Alvarez MT, Bowen J, Carcao MD, Cox Gill J, Key NS, Kouides PA, Kurnik K, Lail AE, Leebeek FW, Makris M, Mannucci PM, Winikoff R, Berntorp E; VWD PN. Prophylaxis in severe forms of von Willebrand's disease: results from the von Willebrand Disease Prophylaxis Network (VWD PN). Haemophilia. 2013 Jan;19(1):76-81. doi: 10.1111/j.1365-2516.2012.02916.x. Epub 2012 Jul 23.
citation: Berntorp E, Abshire T; von Willebrand Disease Prophylaxis Network Steering Committee. The von Willebrand disease prophylaxis network: exploring a treatment concept. J Thromb Haemost. 2006 Nov;4(11):2511-2. doi: 10.1111/j.1538-7836.2006.02179.x. No abstract available.