von Willebrand disease (VWD) is an inherited bleeding disorder, characterised mainly by mucosal bleedings, which may be life-threatening, and joint bleeds in severe VWD cases. VWD is caused by a lack of von Willebrand factor (VWF) and coagulation factor VIII (FVIII). Treatment of VWD aims at normalizing the VWF activity in plasma, which can be achieved by stimulating the endogenous release of VWF with desmopressin (DDAVP, 1-desamino-8-D arginine vasopressin) or by infusion of a VWF concentrate.
Conditions
Interventions
Trade Name: Octostim
Product Name: Octostim
Product Code: DDAVP (Desmopressin)
Pharmaceutical Form: Intravenous infusion
Trade Name: Octostim
Product Name: Octostim
Product Code: DDAVP (Desmopressin)
Sponsors
Rigshospitalet, Copenhagen
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Well-characterized VWD patients with all type 1 and 2 (including 2B if treatment with DDAVP is allowed in any centre), with the widest range of age (from young children to senior adults). Due to the increased number of blood sampling, kinetic studies will be performed in patients older than 12 only. Diagnosis of type 1 and 2 VWD should be correctly performed by the participating centre following recommendations provided by the ISTH-SSC on VWF. If available, molecular diagnosis (mutations) should also be reported to confirm classification in a specific VWD type. In a few patients, to be considered undefined because of a difficult diagnosis, plasma samples should be kept and sent, upon request by the Centre, to a more experienced laboratory. All these difficult diagnosis will be confirmed by the Steering Committee by using clinical and laboratory data. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Patients should be excluded from the study in the following cases: a) Acquired von Willebrand Syndrome; b) Additional congenital and acquired defects of platelet function (ex, Acquired Storage Pool defect + VWD); c) use of anti-inflammatory drugs that may affect platelet function within the previous 10 days; d) previous history of seizures in the family; e) recent serious cardiovascular episodes (Acute myocardial infarction and stroke) in the VWD patient.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To correlate the biological response (and kinetics of FVIII/VWF activities) with clinical efficacy of Desmopressin (DDAVP) in type 1 and 2 VWD patients.;Secondary Objective: To evaluate the biological response in a large number (at least 150, world-wide) of well characterized VWD patients To correlate kinetic data of VWF and FVIII to different subtypes of VWD. To determine the rate of clinical efficacy of DDAVP in different bleeding episodes and during surgery in different subtypes of VWD To evaluate the efficacy of dirrerent modes of administration in clinical practice To register side effects To develop treatment guidelines ;Primary end point(s): The primary end point is the correlation between biological response (maximum levels and kinetics of VWF and FVIII) and clinical response. Criteria for definition of clinical response. The clinical effects of DDAVP will be evaluated by using the following evaluation scale: Excellent: No excessive bleeding; Good: Excess bleeding without need for VWF concentrate treatment; Poor: Excess bleeding with need for VWF concentrate treatment. The loss of blood during surgery will be evaluated directly by the surgeon and indirectly by the actual reduction of haemoglobin. | — |
Countries
Denmark
Outcome results
None listed