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Cord blood effective alternative to matched donor stem cells for kids with rare disorder
Date:3/14/2013

similarly high for children who had received donations of matched sibling cells or fully matched unrelated umbilical cord blood (MSD or UCB, 81%), compared to EFS rates for patients transplanted with cells from matched unrelated donors (66%) or unmatched unrelated cord blood donors (68%). EFS rates were highest among infants who received transplants at age 16 months or younger, indicating the importance of transplantation as early as possible after diagnosis for these children, when treatment may help prevent further disease burden or damage.

Within two months of undergoing transplants, nearly all patients (91%) showed evidence of donor cell recovery (as noted by increasing levels of white blood cells in the blood). Only 12 percent of children experienced secondary graft failure (when the body rejects donor cells).

As is common in patients receiving stem cell transplants, investigators observed reports of graft-versus-host disease (GVHD, which occurs when the donated immune cells attack the patient's cells as foreign tissue) among the patient sample. At 100 days post-transplant, roughly one-fourth of transplant recipients experienced moderate to severe acute GVHD, and only 16 percent experienced chronic GVHD (remaining at five years post-transplant).

After completing their analysis of the children, whose progress had been tracked for approximately five years post transplant, Dr. Boelens' team concluded that the children who received UCB transplants experienced on average a higher rate of donor cell engraftment (complete replacement of the bone marrow by donor cells) and normal enzyme levels than those who had received transplants of matched sibling cells or matched/unmatched unrelated bone marrow stem cells. Nearly all (98%) of the cord blood recipients whose transplanted cells were successfully engrafted had normal enzyme levels, supporting a link between high levels of lysosomal α-L-iduronidase in cord blood and suggested improve
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Contact: Andrea Slesinski
aslesinski@hematology.org
American Society of Hematology
Source:Eurekalert

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