N/A
N=56
Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment
Von Willebrand Diseases
Bottom Line
View on ClinicalTrials.gov: NCT04053699 ↗Enrolled (actual)
56
Serious AEs
0.0%
Results posted
Dec 2023
Primary outcome: Primary: Total Annualized Bleeding Rate (TABR) — 29.13 Bleeding events per year
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Von Willebrand Factor-Containing Product (Drug)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- All
- Sponsor
- Octapharma
- Primary completion
- Jan 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Total Annualized Bleeding Rate (TABR) |
29.13 | — |
| SECONDARY Spontaneous Annualized Bleeding Rate (SABR) |
21.19 | — |
| SECONDARY Consumption of the VWF-containing Product |
77.94 | — |
| SECONDARY Number of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale |
450; 6; 1; 0 | — |
| SECONDARY Number of Surgery With Successful/Unsuccessful Efficacy Assessment |
7; 0; 0; 0 | — |
| SECONDARY Quality of Life (QoL) Assessed Using the Patient-Reported Outcomes Measurement Information System (PROMIS-29) |
47.24; 53.57; 49.96; 49.77; 49.81; 52.78 | — |
| SECONDARY Quality of Life (QoL) Assessed Using a 36-Item Short Form Health Survey, Version 2 (SF-36v2) |
49.87; 49.45; 62.74; 64.03; 62.63; 51.21 | — |
| SECONDARY Quality of Life (QoL) Assessed Using a 10-item Short Form Health Survey (SF-10) |
28.60; 46.56 | — |
| SECONDARY Joint Health Status Assessed Using Hemophilia Joint Health Score (HJHS) |
6.76 | — |
| SECONDARY Menstrual Bleeding Assessed Using Pictorial Blood Loss Assessment Chart (PBAC) Score |
521.7; 279.2; 311.0; 408.8; 289.9; 330.4 | — |
| SECONDARY Number of Participants With Adverse Drug Reactions (ADRs) Associated With Use of Wilate |
— | — |
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.
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT04053699). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.