von Willebrand's disease Haematological Disorders Other coagulation defects
Conditions
Interventions
Treatment details:
The following dosing regimes are only recommendations, based on the Swedish experience with the long-term prophylaxis in VWD:
1. For mucosal, joint bleeds and menorrhagia, the sugg
Sponsors
Octapharma AG (Switzerland)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male and female patients of any age 2. Suffering from congenital VWD 3. In need of replacement therapy with factor concentrate 4. Patients starting with a prophylactic treatment must have documentation of at least three apparently spontaneous bleeding episodes (any bleeding site and treated with factor concentrate) in the 6 months prior to enrolment 5. Patients switching from a prophylactic treatment with another factor concentrate to prophylaxis with Wilate® should have anamnesis of bleeds with respective documentation in the period of 12 months prior to enrolment
Exclusion criteria
Exclusion criteria: 1. Presence of a bleeding disorder other than VWD 2. History of non-compliance 3. Difficulties in achieving venous access that would prohibit prophylaxis 4. Incapability to follow the requirements of the surveillance, e.g. unable to keep a patient diary
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To assess the bleeding frequency in VWD patients prior to and after introduction of regular prophylactic therapy with the VWF-containing concentrate Wilate®. Outcomes will be measured at baseline, 6 and 12 months after treatment. Please note that the number of days the patients missed school or work, as well as occurred adverse drug reactions are documented when the patient visits his doctor, so outcomes may be measured more frequently than every 6 months. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. To describe the joint morbidity prior to and during prophylaxis with Wilate®, using the haemophilia joint health score 2. To monitor absence from school/work prior to and during prophylaxis with Wilate® 3. To evaluate the patient's health-related quality of life prior to and during prophylaxis with Wilate® and the patient's self-reported health status prior to and during prophylaxis with Wilate®, using the validated generic instruments of World Health Organization Quality of Life-BREF (WHOQOL-BREF) (for adults) and the generic children's health-related quality of life (KINDL) (for children), and the disease specific VWD-QoL questionnaire 4. To assess treatment satisfaction and treatment efficacy prior to and during prophylaxis with Wilate®, using a 4-point Verbal Rating Scale (VRS), and the Hemo-SatA treatment questionnaire adapted for VWD (VWD-Sat) 5. To evaluate the tolerability of prophylactic treatment with Wilate®: 5.1. Using a 3-point VRS 5.2. By documenting all possibly related adverse drug reactions by the patients, and 5.3. By assessing and - if applicable - reporting all adverse drug reactions by the treating physician occurring during the treatment period with Wilate® Outcomes will be measured at baseline, 6 and 12 months after treatment. Please note that the number of days the patients missed school or work, as well as occurred adverse drug reactions are documented when the patient visits his doctor, so outcomes may be measured more frequently than every 6 months. | — |
Countries
Germany, Russian Federation, Sweden
Outcome results
None listed