An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment
Заболявания:
Von Willebrand Disease, Type 3
Спонсор: Hoffmann-La Roche
Налично на:
БГ
Обобщение
This non-interventional study (NIS) is designed to collect information on the effectiveness and safety of treatment received in routine clinical care, as well as measure the health-related quality of life (HRQoL) of participants with Type 3 von Willebrand disease (VWD) receiving prophylactic therapy per local standard of care (SOC) over an observation period of at least 24 weeks.
Кой може да участва
Inclusion Criteria:
* Confirmed diagnosis of Type 3 von Willebrand disease (VWD), based on medical records
* Adequate hematologic, hepatic, and renal function
* Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 times weekly, as per prescribed dose) and anticipation to remain on the same regimen during the study
* For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements
Exclusion Criteria:
* Inherited or acquired bleeding disorder other than Congenital Type 3 VWD
* History of gastrointestinal bleeding within 18 months prior to enrollment, or any previous diagnosis of angiodysplasia
* History of intracranial hemorrhage
* Previous or current treatment for thromboembolic disease or signs of thromboembolic disease
* Other conditions (e.g., certain autoimmune diseases) that may increase risk of bleeding or thrombosis
* History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
* Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study, with the exception of anti-retroviral therapy
Интервенции
Von Willebrand Factor Concentrates
DRUG
Von Willebrand Factor Concentrates and Factor VIII Concentrates
DRUG
Factor VIII Concentrates
DRUG
Recombinant Activated Factor VII
DRUG
Activated Prothrombin Complex Concentrate
DRUG
Места на провеждане
27
Белгия (1)
UZ Leuven Gasthuisberg
Leuven
Канада (2)
McGill University Health Center
Montreal , Quebec
The Hospital for Sick Children
Toronto , Ontario
COMPLETED
Colombia (1)
IPS SURA Industriales Medellín
Medellín
Франция (2)
Hopital Claude Huriez - CHU Lille
Lille
Groupe Hospitalier Necker Enfants Malades
Paris
Германия (3)
Universitätsklinikum Bonn
Bonn
Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz
Duisburg
Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin
Frankfurt/M.
Италия (3)
AOU Careggi
Florence , Tuscany
IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan , Lombardy
Universita' Degli Studi La Sapienza-Ist.Di Ematologia