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Umbilical Cord Blood—Biology, Banking, and Therapeutic Use
Transplantation experience over the past 20 years has shown that UCB is
Table 1: Diseases Treatable by Cord Blood Stem Cells
associated with significantly fewer GvHD complications than bone
marrow and peripheral blood transplants. This likely contributes to the
Blood-related disorders
growing use of UCB since chronic GvHD is the leading cause of
Bone marrow failure disorders
59
non-relapse-related mortality in transplantation.
26
A meta-analysis of UCB
Hemoglobinopathies
60
and bone marrow transplantation for malignant and non-malignant
Histiocytic disorders
10
Myelodysplastic/myeloproliferative disorders
61
diseases found similar survival rates but lower GvHD rates for children
Platelet abnormalities
62
and adults, despite more HLA disparity between the UCB transplant
Malignancies
donors and recipients.
27
In HLA-identical sibling donor transplantation,
Leukemias
59
UCB has comparable survival rates but significantly less acute and chronic
Lymphomas
59,63
GvHD than bone marrow.
28
Related UCB transplantation appears to
Plasma cell cancers
64
reduce the risk for GvHD (with reports ranging from 3 to 20% for acute
Bone and soft-tissue sarcomas
59
and from 6 to 14% for chronic) compared with unrelated UCB Solid tumors
31
transplants (with reports ranging from 20 to 50% for acute and from 5
Inherited metabolic and immune disorders
to 30% for chronic).
22
Interestingly, UCB transplant recipients have
Leukodystrophies
23,65
shown higher responses to treatment for chronic GvHD compared with
Lysosomal storage diseases
23
bone marrow and peripheral blood transplant recipients.
29
Peripheral
Severe combined immunodeficiences
10
Other primary immunodeficiences.
10
blood allogeneic transplantations show even higher rates of GvHD
Emerging stem cell applications
compared with bone marrow.
30
Type 1 diabetes
54
Brain injury
56
Autologous hematopoietic stem cell transplants have far fewer
Autoimmune disorders
43
complications due to no risk of immune complications, and are often
Cardiovascular disease
43
indicated in transplant situations when the disease has no genetic
etiology. Indications for autologous transplantation include acute
Table 2: Advantages and Disadvantages of Cord Blood
myeloid leukemia, many forms of non-Hodgkin’s lymphoma, myeloma,
solid tumors, and autoimmune diseases such as multiple sclerosis and Advantages Disadvantages
Crohn’s disease. Autologous cord blood transplants have been Simple harvest with no risk to donor Cell dose
conducted successfully for the treatment of neuroblastoma,
31
aplastic
Long-term storage ability One-time supply
anemia,
32
and lymphoblastic leukemia.
33
As the private UCB banking
Immediately available Potential delayed engraftment time
industry grows, the use of UCB in autologous transplantation is
Immunologically mature
expected to grow as private banks provide readily available
High concentration of stem cells
High proliferative rate
hematopoietic stem cells without complicated harvest for autologous
Younger cells with longer telomeres
transplant indications.
Low incidence of viral contamination
UCB offers several advantages for use in allogeneic and autologous
transplantation over bone marrow and peripheral blood (see Table 2). As donor, locate the donor, establish eligibility, and harvest the cells may result
discussed above, UCB transplants have less GvHD, which allows for a higher in disease relapse or progression precluding transplant feasibility.
3
immune tolerance of HLA disparities in allogeneic transplantation. Siblings
are twice as likely to be able to use one another’s UCB compared with bone One disadvantage of UCB transplantation is low graft cell dose due to the
marrow since UCB transplantation has been performed with one, two, or limited collection volume available. Cell dose affects the time needed for
even three HLA mismatches,
34,35
whereas bone marrow transplantation hematological recovery. Several reports have shown that lower cell doses are
typically requires a perfect HLA match. UCB also has a larger number of CFU- associated with slower engraftment times.
25,34,40,41
Cell dose is a larger
GMs,
36
stem cells with longer telomeres and increased proliferative capacity. obstacle in adult transplantation compared with pediatric transplantation
These characteristics appear to provide a more complete hematological and due to the larger cell doses needed for hematological reconstitution of
immunological reconstitution than bone marrow. A study by Frassoni and larger body masses. Expansion of UCB stem cells and the use of multiple
colleagues found that the bone marrow of children one year post-UCB UCB units are two proposed methods of circumventing the cell dose issue
transplantation had higher numbers of committed and early progenitor cells for adults. Despite the slow engraftment time, one study found that adult
compared with the bone marrow of children one year post-bone marrow recipients of unrelated UCB transplants had similar engraftment, treatment-
transplantation.
37
Additionally, significantly longer telomeres have been related mortality, and disease-free survival rates to those who received
observed in the peripheral blood MNC cells in transplant recipients of UCB related bone marrow or peripheral blood.
42
grafts compared with those receiving peripheral blood grafts, which suggests
a replicative advantage for UCB stem cells.
38
UCB is associated with a lower Emerging Applications
risk for viral contamination compared with bone marrow.
39
The ease and
safety of UCB collection and ability for long-term storage gives UCB the Regenerative Medicine
advantage of immediate availability for autologous and allogeneic clinical UCB stem cells hold promise for regenerative medicine applications to
use, whereas the time needed to identify a bone marrow or peripheral blood treat damaged and diseased cells and tissues outside of the
US OBSTETRICS & GYNECOLOGY 69
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