Von Willebrand Disease (VWD)
Conditions
Keywords
Von Willebrand disease, gastrointestinal bleeding, angiodysplasia, epistaxis
Brief summary
WILL MANAGE is a prospective multicenter controlled open label randomized trial comparing an algorithm-based multidisciplinary management to a standard of care to reduce the incidence of bleeding (GI bleeding or severe epistaxis) recurrence in von Willebrand disease.
Interventions
PROCEDUREOptimized management
systematic gastrointestinal and nasal endoscopic exploration, local endoscopic treatment whenever applicable,prophylaxis with stepwise dose escalation of VWF concentrate and use of antiangiogenic drugs in patients with severe GI angiodysplasia-related bleeding
PROCEDUREStandard of care management
Standard of care management
Sponsors
University Hospital, Lille
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Males and females adults aged \> 18 years * With a constitutional von Willebrand Disease (according to the phenotypic criteria of the French Reference Center of von Willebrand Disease) * With a GI bleeding \[either overt (any hematemesis, any melena or hematochezia with at least a 2g/dL drop in hemoglobin) or occult (iron deficiency anemia with at least 2g/dL drop in hemoglobin)\] with the presence of angiodysplasia or a negative finding on digestive conventional endoscopy OR with a severe epistaxis (requiring red blood cells transfusion or treatment with VWF concentrates) * Be affiliated to a social security scheme * Written informed consent obtained
Exclusion criteria
* Acquired von Willebrand Disease * Presence of an inhibitor to VWF or a contra indication to VWF concentrates * Contra-indication to videocapsule endoscopy * Refusal of the procedures part of the study * Pregnant women or breastfeeding * Short-life expectancy * Liver cirrhosis Child-Pugh C or diagnosed portal hypertension * Cancer currently undergoing chemotherapy * Inability to provide informed consent * Patient under justice protection
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| cumulative incidence of bleeding (GI bleeding or severe epistaxis) recurrence during follow-up | Until the visit 7 (72 months ±2months) | \- GI bleeding is defined as: Any hematemesis or melena Or hematochezia with at least a 2g/dL drop in hemoglobin Or occult bleeding: iron deficiency anemia with at least a 2g/dL drop in hemoglobin \- Severe epistaxis is defined as: A nose bleed requiring red blood cells transfusion or treatment with VWF concentrates |
Secondary
| Measure | Time frame |
|---|---|
| Number of GI bleeding and severe epistaxis recurrence during follow-up | Until the visit 7 (72 months ±2months) |
| Number of days of hospitalization for GI bleeding or severe epistaxis recurrence during follow-up | Until the visit 7 (72 months ±2months) |
| Number of transfused red blood cells units, the total amount of iron supplementation (mg) and number of units of VWF concentrates administred (IU/kg) during follow-up | Until the visit 7 (72 months ±2months) |
| Change in haemoglobin and serum ferritin levels from inclusion to 3-years | from inclusion to 3-years |
| Change in bleeding score (ISTH BAT) from inclusion to 3-years. The bleeding score will be calculated for the 3-year period before the evaluation. | from inclusion to 3-years |
| Change in quality of life assessed by a standardized questionnaire validated for VWD (WISH-QoL scores) from inclusion to 3 years | from inclusion to 3-years |
Countries
France
Contacts
CONTACTMarie Caillierez
PRINCIPAL_INVESTIGATORSophie Susen, MD, Professor
University Hospital, Lille
Outcome results
None listed