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Transplantation outcomes for severe combined immunodeficiency, 2000-2009.

Publication ,  Journal Article
Pai, S-Y; Logan, BR; Griffith, LM; Buckley, RH; Parrott, RE; Dvorak, CC; Kapoor, N; Hanson, IC; Filipovich, AH; Jyonouchi, S; Sullivan, KE ...
Published in: N Engl J Med
July 31, 2014

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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Published In

N Engl J Med

DOI

EISSN

1533-4406

Publication Date

July 31, 2014

Volume

371

Issue

5

Start / End Page

434 / 446

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Transplantation Conditioning
  • T-Lymphocytes
  • Survival Rate
  • Siblings
  • Severe Combined Immunodeficiency
  • Retrospective Studies
  • Retreatment
  • Male
  • Lymphocyte Count
 

Citation

APA
Chicago
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MLA
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Pai, S.-Y., Logan, B. R., Griffith, L. M., Buckley, R. H., Parrott, R. E., Dvorak, C. C., … O’Reilly, R. J. (2014). Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med, 371(5), 434–446. https://doi.org/10.1056/NEJMoa1401177
Pai, Sung-Yun, Brent R. Logan, Linda M. Griffith, Rebecca H. Buckley, Roberta E. Parrott, Christopher C. Dvorak, Neena Kapoor, et al. “Transplantation outcomes for severe combined immunodeficiency, 2000-2009.N Engl J Med 371, no. 5 (July 31, 2014): 434–46. https://doi.org/10.1056/NEJMoa1401177.
Pai S-Y, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, et al. Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med. 2014 Jul 31;371(5):434–46.
Pai, Sung-Yun, et al. “Transplantation outcomes for severe combined immunodeficiency, 2000-2009.N Engl J Med, vol. 371, no. 5, July 2014, pp. 434–46. Pubmed, doi:10.1056/NEJMoa1401177.
Pai S-Y, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Hanson IC, Filipovich AH, Jyonouchi S, Sullivan KE, Small TN, Burroughs L, Skoda-Smith S, Haight AE, Grizzle A, Pulsipher MA, Chan KW, Fuleihan RL, Haddad E, Loechelt B, Aquino VM, Gillio A, Davis J, Knutsen A, Smith AR, Moore TB, Schroeder ML, Goldman FD, Connelly JA, Porteus MH, Xiang Q, Shearer WT, Fleisher TA, Kohn DB, Puck JM, Notarangelo LD, Cowan MJ, O’Reilly RJ. Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med. 2014 Jul 31;371(5):434–446.

Published In

N Engl J Med

DOI

EISSN

1533-4406

Publication Date

July 31, 2014

Volume

371

Issue

5

Start / End Page

434 / 446

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Transplantation Conditioning
  • T-Lymphocytes
  • Survival Rate
  • Siblings
  • Severe Combined Immunodeficiency
  • Retrospective Studies
  • Retreatment
  • Male
  • Lymphocyte Count