Abstract
SY Pai, BR Logan, LM Griffith. N Engl J Med. 2014;371(5):434–446 To evaluate the outcome of hematopoietic stem cell transplantation (HSCT) in patients with severe combined immunodeficiency (SCID) treated in North America between 2000 and 2009 based on data collected by the Primary Immune Deficiency Treatment Consortium. Two hundred forty infants with SCID treated with HSCT at 25 centers during this 10-year period. This was a retrospective study based on data entry into defined forms through the Primary Immune Deficiency Treatment Consortium. Each patient entry was reviewed by 3 content experts and classified as eligible versus not eligible on the basis of defined criteria required to meet the diagnosis of SCID. Transplant information was provided …
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