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

underscore how transplants are becoming an increasingly successful treatment option for more patients with blood cancer than ever before," said Jeffrey Miller, MD, moderator of the press conference and Deputy Director of the Masonic Cancer Center and the Clinical and Translational Sciences Institute at the University of Minnesota in Minneapolis. "Whereas transplant would not have been an option for many patients without a sibling or other perfectly matched donor even several years ago, exciting new strategies have afforded them the opportunity to gain significant benefit from this procedure today. As we are now able to focus our efforts on improving the overall patient experience and reducing the risk of relapse, the leading cause of death after transplant, we have greatly improved long-term survival outcomes for patients who before might not have had another treatment option."

This press conference will take place on Saturday, December 7, 2013, at 10:00 a.m. CST.

Encouraging Outcomes in Older Patients (Pts) Following Nonmyeloablative (NMA) Haploidentical Blood or Marrow Transplantation (haploBMT) With High-Dose Posttransplantation Cyclophosphamide (PT/Cy) [158]

To more fully demonstrate the utility of haploidentical bone marrow or peripheral blood stem cell transplants (haploBMT) as an alternative to fully matched transplants, this study evaluated nonmyeloablative, related haploBMT (with post-transplantation cyclophosphamide) among patients aged 50 75 with poor-risk hematologic malignancies. Results of 273 such transplants performed at Johns Hopkins were compared to determine the impact of older age on outcomes. The two-year probability of progression-free survival was very similar among patients in their 50s, 60s, and 70s (39, 36, and 39%, respectively), as was the two-year probability of overall survival (51, 56, and 44%). Among these age groups, there
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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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