TY - JOUR
T1 - Transplantation outcomes for severe combined immunodeficiency, 2000-2009
AU - Pai, Sung Yun
AU - Logan, Brent R.
AU - Griffith, Linda M.
AU - Buckley, Rebecca H.
AU - Parrott, Roberta E.
AU - Dvorak, Christopher C.
AU - Kapoor, Neena
AU - Hanson, Imelda C.
AU - Filipovich, Alexandra H.
AU - Jyonouchi, Soma
AU - Sullivan, Kathleen E.
AU - Small, Trudy N.
AU - Burroughs, Lauri
AU - Skoda-Smith, Suzanne
AU - Haight, Ann E.
AU - Grizzle, Audrey
AU - Pulsipher, Michael A.
AU - Chan, Ka Wah
AU - Fuleihan, Ramsay L.
AU - Haddad, Elie
AU - Loechelt, Brett
AU - Aquino, Victor M.
AU - Gillio, Alfred
AU - Davis, Jeffrey
AU - Knutsen, Alan
AU - Smith, Angela R.
AU - Moore, Theodore B.
AU - Schroeder, Marlis L.
AU - Goldman, Frederick D.
AU - Connelly, James A.
AU - Porteus, Matthew H.
AU - Xiang, Qun
AU - Shearer, William T.
AU - Fleisher, Thomas A.
AU - Kohn, Donald B.
AU - Puck, Jennifer M.
AU - Notarangelo, Luigi D.
AU - Cowan, Morton J.
AU - O'Reilly, Richard J.
PY - 2014
Y1 - 2014
N2 - Background: The Primary Immune Deficiency Treatment Consortium was formed to analyze the results of hematopoietic-cell transplantation in children with severe combined immunodeficiency (SCID) and other primary immunodeficiencies. Factors associated with a good transplantation outcome need to be identified in order to design safer and more effective curative therapy, particularly for children with SCID diagnosed at birth. Methods: We collected data retrospectively from 240 infants with SCID who had received transplants at 25 centers during a 10-year period (2000 through 2009). Results: Survival at 5 years, freedom from immunoglobulin substitution, and CD3+ T-cell and IgA recovery were more likely among recipients of grafts from matched sibling donors than among recipients of grafts from alternative donors. However, the survival rate was high regardless of donor type among infants who received transplants at 3.5 months of age or younger (94%) and among older infants without prior infection (90%) or with infection that had resolved (82%). Among actively infected infants without a matched sibling donor, survival was best among recipients of haploidentical T-cell - depleted transplants in the absence of any pretransplantation conditioning. Among survivors, reduced-intensity or myeloablative pretransplantation conditioning was associated with an increased likelihood of a CD3+ T-cell count of more than 1000 per cubic millimeter, freedom from immunoglobulin substitution, and IgA recovery but did not significantly affect CD4+ T-cell recovery or recovery of phytohemagglutinin- induced T-cell proliferation. The genetic subtype of SCID affected the quality of CD3+ T-cell recovery but not survival. Conclusions: Transplants from donors other than matched siblings were associated with excellent survival among infants with SCID identified before the onset of infection. All available graft sources are expected to lead to excellent survival among asymptomatic infants. (Funded by the National Institute of Allergy and Infectious Diseases and others.)
AB - Background: The Primary Immune Deficiency Treatment Consortium was formed to analyze the results of hematopoietic-cell transplantation in children with severe combined immunodeficiency (SCID) and other primary immunodeficiencies. Factors associated with a good transplantation outcome need to be identified in order to design safer and more effective curative therapy, particularly for children with SCID diagnosed at birth. Methods: We collected data retrospectively from 240 infants with SCID who had received transplants at 25 centers during a 10-year period (2000 through 2009). Results: Survival at 5 years, freedom from immunoglobulin substitution, and CD3+ T-cell and IgA recovery were more likely among recipients of grafts from matched sibling donors than among recipients of grafts from alternative donors. However, the survival rate was high regardless of donor type among infants who received transplants at 3.5 months of age or younger (94%) and among older infants without prior infection (90%) or with infection that had resolved (82%). Among actively infected infants without a matched sibling donor, survival was best among recipients of haploidentical T-cell - depleted transplants in the absence of any pretransplantation conditioning. Among survivors, reduced-intensity or myeloablative pretransplantation conditioning was associated with an increased likelihood of a CD3+ T-cell count of more than 1000 per cubic millimeter, freedom from immunoglobulin substitution, and IgA recovery but did not significantly affect CD4+ T-cell recovery or recovery of phytohemagglutinin- induced T-cell proliferation. The genetic subtype of SCID affected the quality of CD3+ T-cell recovery but not survival. Conclusions: Transplants from donors other than matched siblings were associated with excellent survival among infants with SCID identified before the onset of infection. All available graft sources are expected to lead to excellent survival among asymptomatic infants. (Funded by the National Institute of Allergy and Infectious Diseases and others.)
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U2 - 10.1056/NEJMoa1401177
DO - 10.1056/NEJMoa1401177
M3 - Article
C2 - 25075835
AN - SCOPUS:84904876386
SN - 0028-4793
VL - 371
SP - 434
EP - 446
JO - New England Journal of Medicine
JF - New England Journal of Medicine
IS - 5
ER -