The characterization of the immunophenotypic profile of PBMC subsets in steady state SCD patients. The determination of general T cell function in SCD patients. Both will be used as reference profiles in further research evaluating immune…
ID
Source
Brief title
Condition
- Haemoglobinopathies
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The characterization of PBMC subsets in SCD patients with the HbSS/HbSß0
genotype as compared to matched healthy controls in order to elucidate
potential perturbations in the adaptive immunity (PBMC subsets) in SCD.
Secondary outcome
The identification of differences between the immunophenotypic profile of PBMC
subsets of adolescent SCD patients compared to matched healthy controls.
The identification of differences between the immunophenotypic profile of PBMC
subsets of adult and adolescent HbSS/HbSß0 genotype SCD patients.
The determination of general T-cell effector function in steady state SCD
patients in order to establish a reference that can be used in further research
evaluation T-cell function after allogeneic SCT for SCD.
Background summary
Sickle cell disease (SCD) is increasingly being recognized as a
pro-inflammatory condition associated with alterations in immune phenotype and
function. These alterations probably contribute to high rates of graft failure
after allogeneic stem cell transplantation (SCT) for SCD. The probability of
graft rejection is generally higher in children and adolescents compared to the
adult SCD population.
While impairments of innate immunity in SCD have been well described, a
profound characterization of subsets of adaptive immune cells in steady state
SCD patients is lacking. Extensive analysis of peripheral blood mononuclear
cell (PBMC) subsets and measurement of general T cell effector function in
steady state SCD patients compared to matched healthy controls will provide an
overview of the alterations of adaptive immune cells present in SCD.
Study objective
The characterization of the immunophenotypic profile of PBMC subsets in steady
state SCD patients. The determination of general T cell function in SCD
patients. Both will be used as reference profiles in further research
evaluating immune reconstitution, graft rejection and T cell function after
allogeneic SCT for SCD.
Study design
This is a cross-sectional laboratory study with a control group. A maximum of
50ml of blood will be drawn from all participants at one time point (T0).
Study burden and risks
For subjects included in this study no direct positive effect can be expected.
The burden for participants will be limited to venipuncture and risks are not
to be expected. This study will provide insight into the immunological
alterations present in SCD patients compared to the healthy population.
Findings of this study will be used as reference for further research involving
SCD patients that undergo or underwent allogeneic SCT.
Meibergdreef 9 9
Amsterdam 1105AZ
NL
Meibergdreef 9 9
Amsterdam 1105AZ
NL
Listed location countries
Age
Inclusion criteria
Inclusion criteria SCD patients
To be eligible to participate in this study, a subject must meet all the
following criteria:
o Age 12 years or older
o High performance liquid chromatography (HPLC) confirmed diagnosis of
HbSS/HbSß0
o Willing and able to provide written informed consent
o For children 12-16 years: additional written informed consent of
parent/caretaker
Inclusion criteria healthy volunteers
To be eligible to participate in this study, a subject must meet all the
following criteria:
o Age 18 years or older
o Willing and able to provide informed consent
o No self-reported medical issues that could interfere with immunity
o Originating (or parents originating) from region where SCD occurs
Exclusion criteria
Exclusion criteria SCD patients
A potential subject who meets any of the following criteria will be excluded
from participation in this study:
o History of allogeneic SCT
o Participation in Amsterdam UMC SCT biobank because of planned allogeneic SCT
o History of chronic infection or autoimmune inflammatory disease
o Pregnancy, self-reported
o Vaso-occlusive disease in the preceding 4 weeks, self-reported
o Red blood cell transfusion in the preceding 3 months, self-reported
Exclusion criteria for healthy volunteers
A potential subject who meets any of the following criteria will be excluded
from participation in this study:
o Carriership of sickle cell gene or other hemoglobinopathy, self-reported
o Chronic infection, self-reported
o Chronic auto-inflammatory disease, self-reported
o Use of chronic medication that affects the immune system, self-reported
Design
Recruitment
Kamer G4-214
Postbus 22660
1100 DD Amsterdam
020 566 7389
mecamc@amsterdamumc.nl
Followed up by the following (possibly more current) registration
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In other registers
| Register | ID |
|---|---|
| CCMO | NL79404.018.21 |