Von Willebrand disease
Conditions
Interventions
Participants enrolled in the prospective phase will undergo an escalation of
treatment from one to three dose levels of VWD product. It is assumed that all
subjects enrolled will begin treatment on
Sponsors
Von Willebrand disease prophylaxis network
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
Inclusion criteria: A. Type 1: eligible for participation if 1.
Exclusion criteria
Exclusion criteria: A. They have acquired VWD;B. They have a history of inhibitory antibodies to VWF;C. They are already on prophylaxis, defined as receiving factor infusions at least once per week to prevent or decrease the severity of bleeding with the intention of maintaining this regimen for, on average, 45 or more weeks per year; or are receiving factor infusions on a regular basis to prevent or decrease the severity of menorrhagia.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Bleeding episodes. The primary outcome for the study is bleeding episodes associated with VWD. Participants will be provided with diaries on which they will prospectively record the date, location, and cause of hemorrhages occurring during the study. Treatment for breakthrough bleeding episodes and prophylactic doses (date, time, dosage) of VWD product will be recorded as well. For the menorraghia study the bleeding will be scored using a standardized score (PBAC). For the arthroscopy study evaluatieon will be performed by a physical therapist using teh WFH PE scale and a pain scale. Radiologic evaluation will be performed using the Petterson score Health related quaity of life will be assessed by the HRQOL-4. | — |
Secondary
| Measure | Time frame |
|---|---|
| Health economics: prospective evaluation of absence of scholl or work product usage hospitalizations laboratory analysis : inhibitors to VWF Safety variables: serious adverse events other adverse events | — |
Countries
Netherlands
Outcome results
None listed