In our laboratory we are able to characterize and culture MSCs. In order to study more extensively the possible alterations in MSC biology in AA we will investigate bone marrow cells of patients with newly diagnosed AA and at the time of response…
ID
Source
Brief title
Condition
- Anaemias nonhaemolytic and marrow depression
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The functional characterization of MSC*s in AA by gene expression profiling and
in vitro behavior
Secondary outcome
Exploratory analysis correlation between response on immunosuppressive therapy
and MSC characteristics at presentation
Background summary
Acquired aplastic anemia (AA) is a hematopoietic stem cell (HSC) disease
associated with bone marrow (BM) failure reflected by markedly reduced
cellularity and deficient blood cell production. The pathophysiology of the
disease is still not completely resolved; however aberrant activation of the
immune system towards the HSC of the patient seems to play a central role. It
is well known that after an inciting event, such as drug exposure or viral
infection, the hematopoietic compartment can be destroyed by the immune system.
Small numbers of surviving stem cells support adequate hematopoiesis for some
time, but eventually the cell counts become very low and symptoms appear. The
attaque on the bone marrow compartment might not only linked to hematopoietic
stem cells but might also be directed to the surrounding microenvironment
including mesenchymal stem cells (MSCs). MSCs play an important role in
providing the specialized bone marrow microenvironment for hematopoietic stem
cell survival and differentiation alterations and defects of these cells in AA
have been described. Furthermore, co-transplantation of haploidentical
mesenchymal stem cells to enhance engraftment of hematopoietic stem cells and
to reduce the risk of graft failure in patients with failure has also been
published supporting the role of MSC in AA.
Study objective
In our laboratory we are able to characterize and culture MSCs. In order to
study more extensively the possible alterations in MSC biology in AA we will
investigate bone marrow cells of patients with newly diagnosed AA and at the
time of response evaluation after immunosuppressive therapy.
Study design
In patients diagnosed with aplastic anemia additional bone marrow cells (20 ml)
will be collected during the standard diagnostic and first evaluation bone
marrow test
Study burden and risks
At the standard procedure of diagnostic and first evaluation bone marrow
punction 20 ml extra marrow is drawn. This increases the procedure with 2
minutes. There are no additional punctions done.
Hanzeplein 1
Groningen 9713GZ
NL
Hanzeplein 1
Groningen 9713GZ
NL
Listed location countries
Age
Inclusion criteria
Patients with suspected a plastic anemia patients undergoing a diagnostic and first evaluation bone marrow-biopsy
Exclusion criteria
Age < 18 years
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 | NL50636.042.14 |