Biomedical Engineering Reference
In-Depth Information
mobilization/harvesting protocol/timing in order to obtain high CD34
+
and especially
a more primitive CD34
+
/CD90
+
cell yield and recovery (using our own controlled-
rate cryopreservation), with critical goal of improving conditions for complete
and long-term hematopoietic reconstitution after autologous SC transplant. The
results obtained in this study clearly confirmed that in sAA (with no allogeneic
donor), autologous SC transplant can result in a complete and long-term medullar
and hematological remission, as well as successful clinical outcome. To our best
knowledge, this was also the second published case of autologous SC transplant in
sAA [
40
] .
Although SC transplant-related mortality and morbidity (TRMM) have decreased,
SC transplants continue to pose multiple potential adverse effects and complica-
tions. The most frequent complications following SC transplant are even now
engrafting failure, infections, and acute or chronic GvHD. Despite the advances
made since the earliest days of transplant therapy, graft failure following allogeneic
SC transplant is still a life-threatening complication. Antibiotic prophylaxis to pre-
vent infections and supportive blood component care to minimize TRMM are criti-
cally important components in management for all, but especially for patients with
chronic GvHD. To decrease GvHD incidence, cell harvest can undergo T-cell deple-
tion and patients are treated prophylactically with a variety of immunosuppressive
drugs. Less toxic transplants, in the form of RIC, are being actively investigated,
with the promise of expanding indications for allogeneic transplants. That is, SC
transplant with RIC can be offered to patients who are disqualified for high-dose
conditioning because of their age or comorbidities. A careful proactive assessment
to identify, treat, and, hopefully, prevent adverse events is essential to a successful
SC transplant.
There is no doubt that SCs are considered an optimal targets for gene therapy.
They are also ideal cells for gene transduction because of their ability to renew
themselves and differentiate into progeny cells and generation of a self-perpetuating
cell population that contains the transduced gene for the lifetime of the patient.
Namely, specific diseases that could be candidates for gene therapy following gene
transduction into SCs as vectors include thalassemia, sickle cell anemia, Fanconi
anemia, purine nucleoside phosphorylase deficiency, chronic granulomatous dis-
ease, leukocyte adhesion deficiency, Gaucher's disease, and a variety of other meta-
bolic deficiencies. UCB-derived SCs potentially could be used to correct genetic
deficiencies at birth after successful gene transduction and autologous transplant.
References
1. Hoffman R (2005) Hematology basic principles and practice, 4th edn. Churchill Livingstone,
New York
2. Balint B, Radovic M, Milenkovic L (1988) Bone marow transplantation. Vojnosanit Pregl
45:195-201
3. Ho AD, Hoffman R, Zanjani ED (2006) Stem cell transplantation. Biology, processing, and
therapy. WILEY-VCH Verlag GmbH & Co KgaA, Weinheim
