Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment
- Conditions
- Von Willebrand Diseases
- Interventions
- Drug: Von Willebrand Factor-Containing Product
- Registration Number
- NCT04053699
- Lead Sponsor
- Octapharma
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 56
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
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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Patients undergoing treatment with a VWF-containing product Von Willebrand Factor-Containing Product Patients with type 3, type 2 (except 2N), or severe type 1 VWD undergoing routine on-demand treatment with a VWF-containing product over a period of 6 months
- Primary Outcome Measures
Name Time Method Total Annualized Bleeding Rate (TABR) Screening through study completion (6 months) The total annualized bleeding rate (TABR) will be calculated as the total number of spontaneous bleeds, traumatic BEs, and other BEs occurring in the time period between the start of data collection for each patient and the Study Completion Visit, divided by the duration (in years) between the start of data collection and the Study Completion Visit. Surgery periods, and BEs occurring within these surgery periods, will be excluded from the calculation of TABR.
- Secondary Outcome Measures
Name Time Method Spontaneous Annualized Bleeding Rate (SABR) Screening through study completion (6 months) Spontaneous annualized bleeding rate (SABR), calculated in analogy with TABR. This includes all bleeding episodes that occurred spontaneously.
Consumption of the VWF-containing Product Screening through study completion (6 months) Data on the consumption of the VWF-containing product (VWF/FVIII IU/kg per month per patient) used for routine on-demand treatment
Quality of Life (QoL) Assessed Using a 10-item Short Form Health Survey (SF-10) At screening QoL assessment based on the results from a SF-10 parent-completed questionnaire for patients ≥5.5 and \<16 years of age, in order to score physical and psychosocial health. SF-10 uses norm-based scoring where scales have a standardized mean value of 50 and standard deviation of 10.
Number of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale Screening through study completion (6 months) The efficacy assessment of bleeding episodes at the end of a BE was evaluated on a 4 point scale by the patient/legal guardian (together with the Investigator in case of on-site treatment) including the four items 'excellent,' 'good,' moderate,' and 'none.' The assessment was excellent when bleeding was completely stopped within 3 days in case of minor bleed, within 7 days in case of major bleed, and within 10 days in case of gastrointestinal bleed; Good when bleeding was completely stopped, but time and/or dose slightly exceeded expectations ; Moderate when bleeding could be stopped only by significantly exceeding time and/or dose expectations; and None when bleeding could be stopped only by using other VWF-containing products.
Quality of Life (QoL) Assessed Using a 36-Item Short Form Health Survey, Version 2 (SF-36v2) At screening visit QoL assessment based on the results from the SF-36v2 questionnaire to measure functional health and well-being in patients ≥16 years. SF-36v2 ranks 8 different domains using a scale standardized with a scoring algorithm to obtain a score ranging from 0 to 100.The eight health domains include physical functioning (PF), role physical (RP), bodily pain (BP), general health problems (GH), vitality (VT), social functioning (SF), role emotional (RE) and general mental health (MH). Higher scores indicate better health status, and a mean score of 50 has been articulated as a normative value for all scales.
Number of Surgery With Successful/Unsuccessful Efficacy Assessment From start of surgery until end of post-operative period (within 8 days after surgery) Effectiveness of VWF-containing product in surgical prophylaxis based on the proportion of surgeries successfully treated. Overall treatment efficacy will be assessed at the end of the postoperative period by the treating physician using predefined criteria of 'Excellent', 'Good', 'Moderate/Poor' or 'None'.
Quality of Life (QoL) Assessed Using the Patient-Reported Outcomes Measurement Information System (PROMIS-29) At screening visit QoL assessment based on the results from the PROMIS-29 survey to monitor and evaluate the physical, mental, and social health in all patients, using a scale of a minimum score of 0 and a maximum score of 10, with higher scores representing a better outcome. The survey covers seven domains from the most relevant areas of self-reported health (depression, anxiety, physical function, pain interference, fatigue, sleep disturbance and ability to participate in social roles and activities) for the majority of people with chronic illness. PROMIS scores have a mean of 50 and standard deviation (SD) of 10 in a referent population. Full details of cut off points for each domain can be found here: https://www.healthmeasures.net/score-and-interpret/interpret-scores/promis/promis-score-cut-points
Joint Health Status Assessed Using Hemophilia Joint Health Score (HJHS) At screening Joint health status will be assessed using the Hemophilia Joint Health Score (HJHS), which has been specifically validated for the assessment of the clinical outcome in VWD. HJHS evaluates six index joints to produce a score between 0-124. Higher scores indicate worse joint health.
Menstrual Bleeding Assessed Using Pictorial Blood Loss Assessment Chart (PBAC) Score Screening through study completion (6 months) Bleeding information from each menstrual period while in this study will be collected using the Pictorial Blood Loss Assessment Chart (PBAC). The PBAC will be provided to all female patients of child-bearing potential. The data documented in the PBAC and the investigator-calculated final score will be recorded in the eCRF. The PBAC records pad and tampon use (as either light \[1 point\], medium \[5 points\], or heavy \[10 points\] flow), clots (small \[1 point\] or large \[5 points\]), and flooding episodes (1 point each) which can be recorded as many times as necessary any day of the month. The PBAC is scored from 0 (no bleeding) onwards, with a score of \>100 defining abnormal coagulation and heavy menstrual bleeding (corresponds to \>80ml of blood loss per menstrual cycle).
Number of Participants With Adverse Drug Reactions (ADRs) Associated With Use of Wilate Screening through study completion (6 months) Noxious and unintended reactions arising from the use of Wilate will be monitored throughout the study.
Trial Locations
- Locations (15)
Debreceni Egyetem Klinikai Központ, Regionális Haemophilia és Thrombophilia Központ
🇭🇺Debrecen, Hungary
American University of Beirut Medical Center
🇱🇧Beirut, Lebanon
"UMHAT Sveta Marina" EAD.
🇧🇬Varna, Bulgaria
University Hospital Centre Zagreb
🇭🇷Zagreb, Croatia
Hotel Dieu de France Hospital
🇱🇧Beirut, Lebanon
Nini Hospital
🇱🇧Tripoli, Lebanon
State Institution "National Children's Specialized Hospital "OKHMATDYT" of the Ministry of Health of Ukraine," Center of Hemostasis Pathology
🇺🇦Kyiv, Ukraine
Community Institution of Lviv Oblast Council "West-Ukrainian Specialized Children's Medical Center
🇺🇦Lviv, Ukraine
Medical Centre Hungarian Defence Forces
🇭🇺Budapest, Hungary
University Clinical Center, Department of Internal Medicine, Hematology
🇭🇺Pécs, Hungary
Specialized Hospital for Active Treatment of Haematological Diseases" EAD, Sofia
🇧🇬Sofia, Bulgaria
Federal State Budgetary Scientific Institution Kirov Scientific-Research Institute of Hematology and Blood Transfusion of Federal
🇷🇺Kirov, Russian Federation
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Republican Research Center for Radiation Medicine and Human Ecology
🇧🇾Gomel, Belarus
Morosovskaya Children Clinical Hospital, Moscow Health Department, Department of General Hematology with the Pathology of Hemostasis
🇷🇺Moscow, Russian Federation