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

(WASHINGTON)-Transplants of blood-forming stem cells from umbilical cord blood may be an effective alternative to transplants of matched donor bone marrow stem cells to treat children with a rare, debilitating disease known as Hurler's syndrome (HS), according to results of a study published online today in Blood, the Journal of the American Society of Hematology (ASH).

HS is an inherited metabolic disease characterized by the lack of a critical metabolic enzyme (lysosomal α-L-iduronidase) that breaks down long chains of sugar molecules in the body. In the absence of the enzyme, the sugar chains build up in the body and damage vital organs. Children with the disease, who can be diagnosed at birth or as late as age 8, typically have a poor prognosis; serious complications such as deafness, halted growth, joint disease, or heart valve problems can rapidly develop after diagnosis and can lead to death. Recently, hematopoietic cell transplants (HCT) have become a valuable treatment for HS, as transplanted cells can generate new, healthy, enzyme producing blood cells to replace the diseased cells. These donor cells will deliver enzymes to all organs, including the brain, and can prevent disease progression. However, clinicians remain challenged to determine the optimal cell source and appropriate conditioning regimens to prepare patients for transplant that will maximize survival and minimize transplant-related morbidity and mortality.

"Cord blood has been proposed as an alternative stem cell source for children with HS since it has been suggested that cord blood may increase their levels of lysosomal α-L-iduronidase, which consequently may allow them to live longer with fewer complications," said lead study author Jaap Jan Boelens, MD, PhD, of the University Medical Center Utrecht, in Utrecht, Netherlands. "However, until now, no studies have comp
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Contact: Andrea Slesinski
aslesinski@hematology.org
American Society of Hematology
Source:Eurekalert

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