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10.1097/ACI.0000000000000115

http://scihub22266oqcxt.onion/10.1097/ACI.0000000000000115
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C4238389!4238389!25259542
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suck abstract from ncbi


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pmid25259542      Curr+Opin+Allergy+Clin+Immunol 2014 ; 14 (6): 521-6
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  • Stem cell transplantation for primary immunodeficiency diseases: The North American Experience #MMPMID25259542
  • Pai SY; Cowan MJ
  • Curr Opin Allergy Clin Immunol 2014[Dec]; 14 (6): 521-6 PMID25259542show ga
  • Purpose of the Review: This review describes recent studies on outcomes after allogeneic hematopoietic cell transplantation (HCT) for primary immunodeficiency (PID) in North America including severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome (WAS) and chronic granulomatous disease (CGD). Recent Findings: Using uniform diagnostic criteria, the Primary Immune Deficiency Treatment Consortium (PIDTC) described the baseline characteristics of newly diagnosed infants with SCID in North America. Analysis of outcomes of HCT for SCID in North America from 2000?2009 showed that young infants, and older infants without active infection, had excellent survival irrespective of type of donor, or transplant approach with regards to conditioning. While pre-transplant conditioning with chemotherapy had a clear and strong negative impact on survival in infants with active infection at the time of transplant, among survivors, conditioning was associated with improved immune reconstitution. However, the potential late effects of conditioning in these infants remain to be characterized. Advances in transplant outcomes for WAS and CGD support the strategy of early transplantation before the onset of severe complications; additional multicenter studies are needed to fully define optimal approaches. Summary: The formation of the PIDTC, a multi-institutional North American consortium, has contributed to our understanding of outcomes after transplant for PID.
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