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International gene therapy trial for 'bubble boy' disease shows promising early results
Date:12/7/2013

the time from donor search request to transplant. Surprisingly, there were no significant differences between the groups in the total number of patients receiving transplants when considering all sources (WNE 63% vs. non-WNE 56%). Compared to WNE patients, non-WNE had more cord blood (21 vs. 3%) and haploidentical transplants (10 vs. 1%), while WNE patients had more fully matched donor transplants (69% found a fully matched donor, while only 20% of non-WNE found the same). Researchers observed a slight difference in wait time from search request to transplant between the two groups (median 110 days for WNE vs. median 132 days for non-WNE); however, the data did not indicate whether the delay would impact outcomes.

"We are encouraged by our results demonstrating that ethnic minority patients' access to transplants has improved considerably, thanks to aggressive recruitment to global registries and data supporting use of new options like haploidentical and cord blood transplants," said study author Robert N. Lown, MD, of University College London Cancer Institute and the Anthony Nolan Research Institute in the United Kingdom. "While we are bridging the gap in access, our next step is to identify potential minority transplant candidates earlier to determine their eligibility and help them seek the right transplant option."

Dr. Lown will present this study during a poster presentation at 5:30 p.m. CST on Saturday, December 7, in Hall G of the Ernest N. Morial Convention Center.

Full-Intensity Transplantation and Short Telomeres Increase the Risk of Cognitive Impairment After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) Results of a Prospective Longitudinal Study [913]

While a hematopoietic stem cell transplant (HSCT) is often a lifesaving procedure, previous reports have associated transplant-related chemotherapy and radiation as having a
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Contact: Irene Sege
irene.sege@childrens.harvard.edu
617-919-3110
Dana-Farber Cancer Institute
Source:Eurekalert  

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International gene therapy trial for 'bubble boy' disease shows promising early results
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