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

ared the safety and efficacy of different stem cell and donor sources among a large group of patients to determine which may be the ideal transplant source."

With this aim, Dr. Boelens and a team of investigators conducted a retrospective analysis analyzing outcomes of HS patients treated with HCT in centers affiliated with the European Group for Blood and Marrow Transplantation (EBMT), Eurocord, and the Center for International Blood and Marrow Transplant Research (CIBMTR). A total of 258 infants and children with HS (ranging from 2 months to 18 years, with a median age of 16 months at transplant) who received an HCT between 1995 and 2007 were selected for analysis based upon certain criteria: a confirmed diagnosis of HS; a transplant of cells from a matched sibling donor (MSD), a matched or mismatched unrelated donor (UD), or cells from a single, unrelated cord blood (UCB) unit; and pre-transplant treatment containing a high dose of chemotherapy to help prevent their immune systems from rejecting donated cells.

Nearly half of the children evaluated in the study (45%) had received transplants of unrelated cord blood (81% of which was from mismatched donors) with the others receiving transplants of bone marrow stem cells from unrelated donors or matched sibling donors. All the patients received pre-transplant high-dose chemotherapy, and 19 percent had previously received intravenous enzyme replacement therapy with lysosomal α-L-iduronidase. Taking into consideration stem cell source and pre-transplant conditioning, the team compared the rates of overall survival (OS), event-free survival (EFS), engraftment (the degree to which transplanted donor cells were able to reproduce into new cells) or graft failure, or death.

The team noted encouraging survival rates among the children with HS who had received an HCT, regardless of source, with 5-year estimated OS and EFS at 74 percent and 63 percent, respectively. Importantly, EFS rates were
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Contact: Andrea Slesinski
aslesinski@hematology.org
American Society of Hematology
Source:Eurekalert

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