The Journal of Pediatrics
Volume 155, Issue 6 , Pages 834-840.e1 , December 2009

Long-term Clinical Outcome of Patients with Severe Combined Immunodeficiency Who Received Related Donor Bone Marrow Transplants without Pretransplant Chemotherapy or Post-transplant GVHD Prophylaxis

  • Mary Dell Railey, MD

      Affiliations

    • Department of Pediatrics, Duke University Medical Center, Durham, NC
    • ,
  • Yuliya Lokhnygina, PhD

      Affiliations

    • Department of Biostatistics and Bioinformatics, Duke University Medical Center, Durham, NC
    • Duke Clinical Research Institute, Duke University Medical Center, Durham, NC
    • ,
  • Rebecca H. Buckley, MD

      Affiliations

    • Department of Pediatrics, Duke University Medical Center, Durham, NC
    • Department of Immunology, Duke University Medical Center, Durham, NC
    • Corresponding Author InformationReprint requests: Dr Rebecca H. Buckley, Box 2898, 363 Jones Building, Duke University Medical Center, Durham, NC 27710.

Received 7 January 2009 ,Revised 8 June 2009 ,Accepted 21 July 2009.

References 

  1. Buckley RH. Molecular defects in human severe combined immunodeficiency and approaches to immune reconstitution. Annu Rev Immunol. 2004;22:625–655
  2. Buckley RH, Schiff SE, Schiff RI, Markert L, Williams LW, Roberts JL, et al. Hematopoietic stem cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med. 1999;340:508–516
  3. Buckley RH, Schiff SE, Sampson HA, Schiff RI, Markert ML, Knutsen AP, et al. Development of immunity in human severe primary T cell deficiency following haploidentical bone marrow stem cell transplantation. J Immunol. 1986;136:2398–2407
  4. Borghans JA, Bredius RG, Hazenberg MD, Roelofs H, Jol-van der Zijde EC, Heidt J, et al. Early determinants of long-term T-cell reconstitution after hematopoietic stem cell transplantation for severe combined immunodeficiency. Blood. 2006;108:763–769
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  8. Neven B, Leroy S, Decaluwe H, Le Deist F, Picard C, Moshous D, et al. Long-term outcome after haematopoietic stem cell transplantation of a single-centre cohort of 90 patients with severe combined immunodeficiency: long-term outcome of HSCT in SCID. Blood. 2009;113:4114–4124
  9. Titman P, Pink E, Skucek E, O'Hanlon K, Cole TJ, Gaspar J, et al. Cognitive and behavioural abnormalities in children following haematopoietic stem cell transplantation for severe congenital immunodeficiencies. Blood. 2008;112:3907–3913
  10. Patel NC, Chinen J, Rosenblatt HM, Hanson IC, Brown BS, Paul ME, et al. Long-term outcomes of nonconditioned patients with severe combined immunodeficiency transplanted with HLA-identical or haploidentical bone marrow depleted of T cells with anti-CD6 mAb. J Allergy Clin Immunol. 2008;122:1185–1193
  11. Myers LA, Patel DD, Puck JM, Buckley RH. Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival. Blood. 2002;99:872–878
  12. Sarzotti-Kelsoe M, Win CM, Parrott RE, Cooney M, Moser BK, Roberts JL, et al. Thymic output, T cell diversity and T cell function in long-term human SCID chimeras. Blood. 2009;114:1445–1453
  13. Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med. 2009;360:447–458
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  21. Haddad E, Deist FL, Aucouturier P, Cavazzana-Calvo M, Blanche S, Basile GD, et al. Long-term chimerism and B-cell function after bone marrow transplantation in patients with severe combined immunodeficiency with B cells: a single-center study of 22 patients. Blood. 1999;94:2923–2930

 Supported by grants from the National Institute of Allergy and Infectious Diseases (AI042951 and AI47605 ) and a grant from the National Center for Research Resources (NCRR) (#1UL1 RR024128-01), a component of the National Institutes of Health (NIH) and NIH Roadmap for Medical Research. The authors declare no conflicts of interest.

PII: S0022-3476(09)00747-1

doi: 10.1016/j.jpeds.2009.07.049

The Journal of Pediatrics
Volume 155, Issue 6 , Pages 834-840.e1 , December 2009

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