Bleeding Disorder of Unknown Cause in the Netherlands
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age12+
SponsorMaastricht University Medical Center
About this trial
The purpose of the Bleeding Disorder of Unknown Cause in the Netherlands study (BDUC-iN) is to learn more about unexplained bleeding in individuals with a bleeding disorder of unknown cause (BDUC). The study aims to better understand why these individuals have increased bleeding and how it affects their health and daily life.
The main questions of this study are:
1. What are the mechanisms underlying the bleeding tendency in BDUC? 2. How do bleeding symptoms affect patients' daily functioning and overall health-related quality of life? 3. How is care delivered to individuals with BDUC, and how can this be improved?
Participants with increased bleeding tendency who remain undiagnosed after standard coagulation testing and are consequently classified as having BDUC will be enrolled across the Hemophilia treatment centers in the Netherlands. Participants will undergo blood sampling for advanced hemostasis testing and genetic analysis. In addition, participants will complete validated questionnaires to assess bleeding symptoms and health-related quality of life. Participants will be followed longitudinally to evaluate how bleeding symptoms affect daily activities, medical procedures, and overall health-related quality of life.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Referred to a (pediatric) hemostasis specialist for evaluation of bleeding tendency.
Increased bleeding tendency based on: Abnormal International Society on Thrombosis and Haemostasis Bleeding Assessment Tool (ISTH-BAT) score (≥ 5 in women age 18-30; ≥ 6 in women age 31-51, ≥ 7 in women age 52 or older; ≥4 in men and ≥ 3 in children) OR Clinical gestalt according to the investigating physician
Complete blood count: Hemoglobin > 6.0 mmol/L; thrombocyte count > 100 x10^9/L
Prothrombin (PT) and activated Partial Thromboplastin Time (aPTT): within local reference range, or prolonged without explanatory factor deficiency
Disqualifiers
Use of medication interfering with laboratory hemostasis tests which cannot be stopped before blood withdrawal
Pregnancy or lactation at moment of inclusion
Presence of an established bleeding disorder
Presence of an acquired cause or another explanation for the increased bleeding tendency
Trial population
Individuals aged ≥12 years with a clinically relevant increased bleeding tendency, based on an elevated ISTH Bleeding Assessment Tool (ISTH-BAT) score or the clinical view of a (pediatric) hemostasis specialist, who remain undiagnosed following comprehensive laboratory investigation and are consequently classified as having a bleeding disorder of unknown cause (BDUC), will be identified at one of the six Hemophilia treatment centers in the Netherlands and approached for study participation by their treating physician.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
500 Participants
are grouped into 1 trial groupGroup A: Bleeding Disorder of Unknown Cause
Trial outcomes
Primary outcomes
1
Health-related quality of life
Using the Patient-Reported Outcomes Measurement Information System (PROMIS) in adults and the Pediatric Quality of Life Inventory (PedsQL) in children. PROMIS: responses are converted into T-scores, where higher scores indicate a greater level of the concept being measured (e.g., higher physical function or greater pain, depending on the domain). PedsQL: range 0-100 scale, with higher scores indicating better HRQoL.
Time frame
From enrollment to the end at 10 years2
Diagnostic yield
The diagnostic yield of advanced platelet function, hemostasis and fibrinolytic testing in individuals with BDUC, by examining the frequency and nature of the identified hemostasis abnormalities and their potential relevance to the bleeding phenotype.
Time frame
At baselineSecondary outcomes
1
Bleeding symptoms
Prevalence of bleeding symptoms in patients with BDUC.
Time frame
From enrollment to the end at 10 years2
Bleeding severity
Bleeding severity, using the International Society on Thrombosis and Haemostasis Bleeding Assessment Tool (ISTH-BAT). Range 0 to 56, with higher scores indicating a greater number and/or severity of bleeding symptoms.
Time frame
From enrollment to the end at 10 years3
Physical activity
Physical activity using the Short Questionnaire to Assess Health-Enhancing Physical Activity (SQUASH). Responses are converted into a total activity score, with higher scores indicating higher levels of physical activity.
Time frame
From enrollment to the end at 10 years4
Patient activation
Patient activation measured using the Short Questionnaire to Assess Health-Enhancing Physical Activity (SQUASH). Responses are converted into a total activity score, with higher scores indicating higher levels of physical activity.
Time frame
From enrollment to the end at 10 yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Maastricht University Medical Center
Lead sponsor
Sanquin Research & Blood Bank Divisions
Collaborator
Source ClinicalTrials.gov