The objective of this study is to investigate if CEC are elevated in SCD (and if they could therefore serve as a potential tool for assessing SCD activity), and what the relation of CEC is to the patients medical history and to specific laboratory…
ID
Source
Brief title
Condition
- Haemoglobinopathies
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
1. Comparison of the number of CEC in sickle cell patients to the number in
healthy controls.
Secondary outcome
2. To study the relationship of CEC to the history of clinical vaso-occlusion
(e.g., the frequency of painful crises).
3. To analyze the relationship of CEC to established organ damage and
dysfunction.
4. To determine the relationship of CEC to laboratory risk factors for poor
outcome (e.g., the degree of anemia and the percentage of fetal haemoglobin).
5. To analyze the relationship of CEC to other biomarkers of disease activity
(e.g. endothelial activation and fucntion, the hypercoagulable state and
angiogenesis.
Background summary
Sickle cell disease Sickle cell disease (SCD) is the most important
hemoglobinopathy worldwide, characterized by chronic hemolysis, increased
susceptibility to infections and recurrent vaso-occlusive events that culminate
in significant ischemic organ damage resulting in a diminished quality of life
and early death. A major problem in the management of SCD is lack of objective
tools to accurately assess the extent of the ongoing vaso-occlusive process. An
objective laboratory tool to monitor sickle cell vaso-occlusion is paramount
as, in most sickle cell patients, the frequency of acute vaso-occlusive events
that require medical care correlates poorly to the ischemic end organ damage
characteristic of most sickle cell patients. Ischemic organ damage in SCD
develops as a result of ongoing vascular insults initiated by the adhesion of
sickle red cells and activated neutrophils to the activated vascular
endothelium. Therefore, accurately monitoring ongoing vascular damage could not
only help in identifying patients at risk for developing severe organ damage
but could also serve as a tool to study the effect of potential therapeutics in
clinical trials. Currently, assissing the number of circulating endothelial
cells (CEC) in the blood is considered a promising marker of vasculopathy in
diseases ranging from atherosclerosis to systemic vasculitis.
Study objective
The objective of this study is to investigate if CEC are elevated in SCD (and
if they could therefore serve as a potential tool for assessing SCD activity),
and what the relation of CEC is to the patients medical history and to specific
laboratory parameters (such as the percentage of fetal hemoglobin and soluble
markers of endothelial activation).
Study design
Case-control study
Study burden and risks
The potential risks and burden are minimal, as extra blood (a total of 29
milliliters) will be collected preferably at the moment when blood is being
drawn anyway for regular check-ups. Healthy blood donors serve as controls and
extra blood will be drawn from them at the moment of blood donation.
Postbus 2040
3000 CA, Rotterdam
Nederland
Postbus 2040
3000 CA, Rotterdam
Nederland
Listed location countries
Age
Inclusion criteria
1. High performance liquid chromatography confirmed diagnosis of HbSS or HbSb0-thalassemia genotype.
2. Written informed consent by the patient.
3. For healthy controls: HbAA genotype, race sex and age matched blood donors, written informed consent.
Exclusion criteria
1. Blood transfusion in the preceding three months.
2. Painful crisis/acute chest syndrome/stroke or other acute complications in the preceding 2 weeks.
3. Pregnancy, active cancer, active infection, connective tissue diseases.
4. For healthy controls: any current disease
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 | NL14742.078.06 |