Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment
United States, Belarus, Bulgaria56 participantsStarted 2019-06-25
Plain-language summary
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.
Who can participate
Age range66 Months
SexALL
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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 …
What they're measuring
1
Total Annualized Bleeding Rate (TABR)
Timeframe: Screening through study completion (6 months)