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February 10, 2015

Telomere Length in Hematopoietic Stem Cell Transplantation for Severe Aplastic AnemiaIs It Ready for “Prime Time”?

Author Affiliations
  • 1Blood and Marrow Transplantation & Cell Therapy Program, Birmingham, Alabama

Copyright 2015 American Medical Association. All Rights Reserved. Applicable FARS/DFARS Restrictions Apply to Government Use.

JAMA. 2015;313(6):571-572. doi:10.1001/jama.2015.8

Severe aplastic anemia is a heterogeneous bone marrow failure syndrome, characterized by peripheral blood pancytopenia and bone marrow aplasia. The disease affects both children and young adults. The incidence is estimated at 2 per 1 million annally.13 Early treatment may determine patient outcomes, thus developing better treatments for this condition could have an important clinical implications. Immunosuppression and use of some newer drugs have been shown to be effective.46 However, allogeneic hematopoietic stem cell transplant (HSCT) can be curative for severe aplastic anemia.79 Although the survival results of HLA-matched related donor and HLA-matched unrelated donor HSCT in leukemia are comparable, this is not the case in severe aplastic anemia.10 The results of matched unrelated donor HSCT for severe aplastic anemia have been suboptimal. Compared with 80% to 90% expected long-term survival with matched related donor HSCT, that of matched unrelated donor HSCT has been 39% to 64%.10 Thus, further improvement of the survival results in matched unrelated donor HSCT in severe aplastic anemia is critically important; thus, risk factors associated with improved outcome need to be identified.

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