Biomedical Engineering Reference
In-Depth Information
Multiple myeloma.
Multiple myeloma (MM) is a plasma cell disorder with an
incidence of 4-5 cases per 100,000/year. It remains incurable with conventional
therapies. New therapeutic agents like bortezomib as inhibitor of NFkappaB,
immunomodulatory drugs, and angiogenesis inhibitors improved the disease
course with an increased overall response rate ³ 80% [
71
]. HDT followed by autol-
ogous SC transplant prolonged OS in comparison to standard therapy resulted in
prolongation of the OS more than 10 years. This approach presents standard of
care for younger patients with MM. Regarding tandem use of autologous SC
transplant, its use will decrease, mainly due to the thalidomide maintenance [
72
] .
In high-risk MM patients including cytogenetic [t(4; 14), t(14; 16), t(14; 20), del
p53, del 13, hypodiplody, complex karyotype] or those with progressive disease
during induction therapy, the use of novel agents as bortezomib improved the
clinical course of disease. In high-risk patients, induction therapy followed by
autologous SC transplant plus allo-RIC represents an adequate clinical approach.
Aplastic Anemia
Hypoplasia or aplasia of BM and extreme reduction of polymorphonuclear count in
circulation are typical parameters for categorization of aplastic anemia (AA): very
severe AA < 0.2 × 10
9
/L, severe AA (sAA) 0.2-0.5 × 10
9
/L, and mild AA > 0.5 × 10
9
/L.
Research data confirmed immune-mediated etiology of disease: (a) reduced
hematopoietic progenitor cell quantity in BM; (b) elevated T cell count and inter-
feron gamma (IFN-g) production; and (c) altered mesenchymal cell function,
expressed in a decreased inhibitory effect upon T cells [
40,
73-75
] . The standard
therapeutic approaches for AA are the use of immunosuppressive therapy - IST
(mainly for older individuals) or allogeneic SC transplant (primarily for younger
patients). Usually BM is the source of allogeneic SCs and cells can be collected in
the steady-state hematopoiesis or following BM-priming (BM-activation) using
rHuGM-CSF or rHuG-CSF in low doses [
1-
4,
36,
37
] . Syngeneic bone marrow
transplant in patients with severe aplastic anemia is rare, and usually requires pre-
transplant conditioning to provide engraftment. We report on a patient with hepati-
tis-associated sAA who was successfully treated with syngeneic PB-derived SC
transplant after a series of infectious and bleeding complications. This is the second
case of successful syngeneic SC transplant in hepatitis-associated sAA in the avail-
able literature [
40
] .
The potential treatment by autologous SC transplant in sAA is still an innova-
tive/pioneering therapeutic approach. Namely, majority of patients with sAA have
residual hematopoietic SCs and could be successfully mobilized following initial
IST and extended administration of rHuG-CSF [
75
] . The collected and cryopre-
served cell could be afterwards clinically applied, that is, reinfused, for the treat-
ment of sAA.
Finally, we have presented experience with sAA patient, successfully treated by
autologous SC transplant [
40
]. The objective of our work was to optimize the
