To characterise the different platelet activating pathways using the FACS assay and the different steps of primary hemostasis with a perfusion flow model in patients with hereditary thrombocytopathies; Storage Pool Disease (SPD), Glanzmann*s…
ID
Source
Brief title
Condition
- Platelet disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
1) Platelet reactivity with increasing concentrations of agonist with or
without an added constant concentration of antagonist. Activation is measured
with Fluorescence Activated Cell Sorting (FACS) analysis and expressed in
percentage positive, Median Fluorescence Intensity (MFI), the concentration of
agonist which leads to half maximum effect (EC50) and Area Under the dose
response Curve (AUC).
2) Platelet aggregate formation measurements will be performed using a
perfusion flow model. The main steps in platelet aggregate formation (adhesion,
aggregation and spreading) will be studied and quantified.
Secondary outcome
The secondary parameter/outcome is to study the correlation between the newly
developed the ISTH/SSC Bleeding Assessment Tool and outcomes of FACS and
Perfusion measurements.
Background summary
The routine analysis of coagulation in a patient with a suspected (primary)
hemostatic bleeding disorder is currently limited to a series of mostly static
screening methods. It is estimated that with this current screening method, the
cause of the bleeding disorder is not found in 25% to 60% of patients. Two
novel methods of platelet function analysis in both static and dynamic
conditions were developed and will applied in a group of patients with a well
described bleeding disorder; hereditary thrombocytopathies. If the instruments
are able to identify the pathological pathways involved in the diseases, they
might earn a spot in the algorithm of screening for the evaluation of bleeding
disorder of unknown aetiology in the future. With the aim to increase the
percentage of identified causes of bleeding disorders.
Study objective
To characterise the different platelet activating pathways using the FACS assay
and the different steps of primary hemostasis with a perfusion flow model in
patients with hereditary thrombocytopathies; Storage Pool Disease (SPD),
Glanzmann*s thrombasthenia and Bernard-Soulier syndrome (BSS).
Study design
Monocenter observational cohort study, patients are invited for one visit to
the UMC Utrecht for a venepuncture and a bleeding symptoms questionnaire. No
alterations in lifestyle or use of prescribed medications will be made.
Study burden and risks
Two novel methods of platelet function analysis will be studied in patients
with hereditary thrombocytopathies. This might lead to the introduction of new
diagnostic methods for the screening of patients with a suspected bleeding
disorder in the future. Patients will pay one (extra) visit to the UMC Utrecht
for a venepuncture with a total amount of 31.5ml of blood and a questionnaire
on bleeding symptoms. The results of this study will not be directly beneficial
for the participating patients. Adverse events are not expected to occur in
this study.
Heidelberglaan 100
3584 CX, Utrecht
NL
Heidelberglaan 100
3584 CX, Utrecht
NL
Listed location countries
Age
Inclusion criteria
1) Confirmed diagnosis of Storage Pool Disease or other hereditary thrombocytopathy.
2) 18 years of age or older
Exclusion criteria
1) Uncertainty about diagnosis expressed by their treating physician.
2) Use of medication that is known to influence platelets such as acetylsalicylic acid,
clopidogrel, dipyridamole and NSAIDs (Non-Steroidal Anti-Inflammatory Drugs) within 14 days prior to inclusion.
3) Younger than the age of 18.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL37987.041.11 |