title: Transplantation outcomes for severe combined immunodeficiency, 2000-2009.
abstract: 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.).
authors: The authors' affiliations are listed in the Appendix.
authors: Sung-Yun
authors: SY
authors: Pai
authors: Brent R
authors: BR
authors: Logan
authors: Linda M
authors: LM
authors: Griffith
authors: Rebecca H
authors: RH
authors: Buckley
authors: Roberta E
authors: RE
authors: Parrott
authors: Christopher C
authors: CC
authors: Dvorak
authors: Neena
authors: N
authors: Kapoor
authors: Imelda C
authors: IC
authors: Hanson
authors: Alexandra H
authors: AH
authors: Filipovich
authors: Soma
authors: S
authors: Jyonouchi
authors: Kathleen E
authors: KE
authors: Sullivan
authors: Trudy N
authors: TN
authors: Small
authors: Lauri
authors: L
authors: Burroughs
authors: Suzanne
authors: S
authors: Skoda-Smith
authors: Ann E
authors: AE
authors: Haight
authors: Audrey
authors: A
authors: Grizzle
authors: Michael A
authors: MA
authors: Pulsipher
authors: Ka Wah
authors: KW
authors: Chan
authors: Ramsay L
authors: RL
authors: Fuleihan
authors: Elie
authors: E
authors: Haddad
authors: Brett
authors: B
authors: Loechelt
authors: Victor M
authors: VM
authors: Aquino
authors: Alfred
authors: A
authors: Gillio
authors: Jeffrey
authors: J
authors: Davis
authors: Alan
authors: A
authors: Knutsen
authors: Angela R
authors: AR
authors: Smith
authors: Theodore B
authors: TB
authors: Moore
authors: Marlis L
authors: ML
authors: Schroeder
authors: Frederick D
authors: FD
authors: Goldman
authors: James A
authors: JA
authors: Connelly
authors: Matthew H
authors: MH
authors: Porteus
authors: Qun
authors: Q
authors: Xiang
authors: William T
authors: WT
authors: Shearer
authors: Thomas A
authors: TA
authors: Fleisher
authors: Donald B
authors: DB
authors: Kohn
authors: Jennifer M
authors: JM
authors: Puck
authors: Luigi D
authors: LD
authors: Notarangelo
authors: Morton J
authors: MJ
authors: Cowan
authors: Richard J
authors: RJ
authors: O'Reilly
mesh_terms: D017252:CD3 Complex / Q000097:blood; D005260:Female; D006086:Graft vs Host Disease / Q000453:epidemiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000401:mortality; D006801:Humans; D007070:Immunoglobulin A / Q000097:blood; D015994:Incidence; D007223:Infant; D018655:Lymphocyte Count; D008297:Male; D019233:Retreatment; D012189:Retrospective Studies; D016511:Severe Combined Immunodeficiency / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D035781:Siblings; D015996:Survival Rate; D013601:T-Lymphocytes / Q000276:immunology; D019172:Transplantation Conditioning; D016896:Treatment Outcome
chemical_list: D017252:CD3 Complex; D007070:Immunoglobulin A