A Prospective, Multicenter, Non-Interventional Study Evaluating the Bleeding Incidence in Patients With Von Willebrand Disease Undergoing On-Demand Treatment

WIL-29

The purpose of this study is to prospectively obtain reliable data on the bleeding and treatment pattern of patients with VWD undergoing on-demand treatment with a VWF-containing product over a period of 6 months. The data obtained will be used as a basis for historical comparisons with the bleeding and treatment pattern obtained from a clinical study on the efficacy of prophylactic treatment with a VWF/FVIII concentrate.

2019-06-25

2021-01-31

2021-01-31

Completed

56

Inclusion Criteria: Patients who meet all of the following criteria are eligible for the study: * Male or female patients aged ≥5.5 years at the time of enrolment * VWD type 1 (baseline von Willebrand factor activity \[VWF:RCo\], \<30 IU/dL), 2A, 2B, 2M, or 3 according to medical history requiring substitution therapy with a VWF-containing product to control bleeding * Currently receiving frequent on-demand treatment with a VWF-containing product * In female patients of child-bearing potential using hormonal contraception, the medication class should remain unchanged for the duration of their study participation * Voluntarily given, fully informed written and signed consent obtained before collection of any patient data Exclusion Criteria: Patients who meet any of the following criteria are not eligible for the study: * Patients currently on prophylaxis for VWD (except for perioperative prophylaxis) as well as patients having received treatment once a month for menstrual bleeding, but not for any other bleeds * Patients whose VWD treatment is planned to be switched from on-demand to prophylactic treatment in the next 6 months * History, or current suspicion, of VWF or FVIII inhibitors * Medical history of a thromboembolic event within 6 months before enrolment * Severe liver or kidney diseases as described in the medical records * Female patients with an existing or suspected pregnancy or who are breast-feeding at the time of enrolment * Change in hormonal contraception within 6 months before enrolment * Cervical or uterine conditions causing abnormal uterine bleeding (including infection or dysplasia) * Other coagulation disorders or bleeding disorders due to anatomical reasons * Participation in an interventional clinical study during the 6-month of study period * Inability to complete the patient diary to reliably evaluate the type, frequency, and treatment of BEs during the 6-month study period

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'count': 56, 'type': 'ACTUAL'}}

2023-12-07