Von Willebrand Disease
Conditions
Keywords
vWD, von Willebrand Factor, vWF
Brief summary
Description of the long-term evolution of patients with von Willebrand disease and treated with Voncento® and of the hemostatic efficacy in the prevention and the treatment of non-surgical bleeding episodes and prevention of surgical bleeding during 2 years after patient inclusion.
Detailed description
Inherited von Willebrand disease (VWD) is considered the most common bleeding disorder. Its prevalence is approximately 1% in the general population but symptomatic patients are rarer (0.01%). It is caused by a partial or total quantitative deficiency (type 1 and type 3) or by a qualitative defect (type 2) of von Willebrand factor (VWF), a large multimeric protein that is required for platelet adhesion and serves as factor VIII (FVIII) carrier. Type 2 VWD is further divided in four subgroups (2A, 2B, 2M, and 2N) that are distinguished according to the nature of the VWF defect. Most patients with type 1 VWD can be treated with the synthetic vasopressin analogue desmopressin (DDAVP; 2-desamino-8-D-arginine vasopressin), whereas patients with type 3 VWD and most patients with type 2 VWD require concentrates containing VWF. Plasma-derived FVIII concentrates, which were initially developed for the treatment of haemophilia, contain large amounts of VWF and are used in patients for whom DDAVP treatment is deemed ineffective or contraindicated. Voncento® (CSL Behring) is a plasma-derived FVIII/VWF concentrate registered in France since 2015 for the treatment and prevention of bleeding events in patients with inherited VWD. OPALE is an observational study describing the use of human coagulation FVIII/VWF concentrate (Voncento®) to treat and prevent bleeding episodes in a French cohort of patients with inherited von Willebrand disease in the real life settings. The aim of the OPALE study is to describe the efficacy and the safety of Voncento® in the prophylaxis and treatment of haemorrhage or surgical bleeding.
Interventions
BIOLOGICALVoncento®
Solution for injection
Sponsors
CSL Behring
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Patients suffering from constitutional von Willebrand disease for whom Desmopressin treatment is deemed ineffective or contraindicated * Patients suffering from von Willebrand disease and being treated or having been treated with Voncento® for the treatment of surgical and non-surgical bleeding episodes, the prophylaxis of surgical and non-surgical bleedings * Patients with no history or suspicion of inhibitors (judged on previous efficacy)
Exclusion criteria
* Refusal of the patient or the patient's legal representative to take part in the study; * Existence of a contraindication to the use of Voncento® treatment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Global assessment by the patient and the investigator of the hemostatic efficacy of Voncento® in the management of non-surgical bleeding episodes | Up to 24 months |
| Number of non-surgical bleeding episodes per year | Up to 24 months |
| Number of administrations of Voncento® needed to treat a non-surgical bleeding episode and for the long term prophylaxis | Up to 24 months |
| Total dose of Voncento® (in IU/kg of VWF) needed to treat a non-surgical bleeding episode and for the long term prophylaxis | Up to 24 months |
Secondary
| Measure | Time frame |
|---|---|
| Assessment by the investigator of the hemostatic efficacy of Voncento® during the treatment and the prophylaxis of surgical bleedings and after surgical procedures | Up to 24 months |
| Collection of available biological data (ex: FVIII, VWF:Rco, VWF:Ag) | At baseline and up to 24 months |
| Number of administrations of Voncento® needed to prevent or treat surgical bleeding episode | Up to 24 months |
| Total dose of Voncento® (in IU/kg of VWF) needed to prevent or treat surgical bleeding episode | Up to 24 months |
| Nature and impact of adverse events and in particular serious adverse events, adverse events related to Voncento® | Up to 24 months |
Countries
France
Outcome results
None listed