Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Case Report
  • Published:

Successful unrelated cord blood transplantation in two children with severe combined immunodeficiency syndrome

Bone Marrow Transplantation volume 31, pages 133–136 (2003)Cite this article

Summary:

We describe the successful unrelated cord blood transplantation in two patients affected by a Zap-70 deficiency and an Omenn-like syndrome, respectively. The patients were hospitalised for recurrent infections at the age of 13 and 2 months, respectively. An unrelated cord blood unit was found for each. The conditioning regimen was cyclophosphamide, busulfan and antithymocyte globulin. The total number of infused cells was 15.1×107/kg and 17×107/kg, respectively. Neutrophil engraftment was achieved on days +15 and +23, and platelet count >50×109/l was achieved on days +21 and +52, respectively. One patient presented acute Graft-versus-host disease (GVHD) grade I and the other grade III. Chimerism was mixed and full donor. Normal lymphoproliferative response to mitogens and alloantigens was detectable at 6 months for both. No chronic GVHD was observed in either. The patients are alive and well at 53 and 15 months after transplantation. In conclusion, umbilical cord blood represents a valid alternative source of haemopoietic stem cells.

This is a preview of subscription content, access via your institution

Access options

Buy this article

  • Purchase on Springer Link
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

Similar content being viewed by others

References

  1. Rosen FS, Cooper MD, Wedgwood RJ . The primary immunodeficiencies. N Engl J Med 1995; 333: 431–440.

    Article  CAS  Google Scholar 

  2. Gatti RA, Meuwissen HJ, Allen HD et al. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet 1968; 2: 1366–1369.

    Article  CAS  Google Scholar 

  3. Haddad E, Landais P, Friedrich W et al. Long-term immune reconstitution and outcome after HLA-nonidentical T-cell-depleted bone marrow transplantation for severe combined immunodeficiency: a European retrospective study of 116 patients. Blood 1998; 91: 3646–3653.

    CAS  Google Scholar 

  4. Buckley RH, Schiff SE, Schiff RI et al. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med 1999; 340: 508–516.

    Article  CAS  Google Scholar 

  5. Amrolia P, Gaspar HB, Hassan A et al. Nonmyeloablative stem cell transplantation for congenital immunodeficiencies. Blood 2000; 96: 1239–1246.

    CAS  Google Scholar 

  6. Toren A, Nagler A, Amariglio N et al. Successful human umbilical cord blood stem cell transplantation without conditioning in severe combined immune deficiency. Bone Marrow Transplant 1999; 23: 405–408.

    Article  CAS  Google Scholar 

  7. Knutsen AP, Wall DA . Umbilical cord blood transplantation in severe T-cell immunodeficiency disorders: two-year experience. J Clin Immunol 2000; 20: 466–476.

    Article  CAS  Google Scholar 

  8. Niehues T, Rocha V, Filipovich AH et al. Factors affecting lymphocyte subset reconstitution after either related or unrelated cord blood transplantation in children – a Eurocord analysis. Br J Haematol 2001; 114: 42–48.

    Article  CAS  Google Scholar 

  9. Moretta A, Maccario R, Fagioli F et al. Analysis of immune reconstitution in children undergoing cord blood transplantation. Exp Hematol 2001; 29: 371–379.

    Article  CAS  Google Scholar 

  10. Elder ME, Skoda-Smith S, Kadlecek TA et al. Distinct T cell developmental consequences in humans and mice expressing identical mutations in the DLAARN motif of ZAP-70. J Immunol 2001; 166: 656–661.

    Article  CAS  Google Scholar 

  11. Chilosi M, Facchetti F, Notarangelo LD et al. CD30 cell expression and abnormal soluble CD30 serum accumulation in Omenn's syndrome: evidence for a T helper 2-mediated condition. Eur J Immunol 1996; 26: 329–334.

    Article  CAS  Google Scholar 

  12. Aleman K, Noordzij JG, de Groot R et al. Reviewing Omenn syndrome. Eur J Pediatr 2001; 160: 718–725.

    Article  CAS  Google Scholar 

  13. Rubinstein P, Dobrila L, Rosenfield RE et al. Processing and cryopreservation of placental/umbilical cord blood for unrelated bone marrow reconstitution. Proc Natl Acad Sci USA 1995; 92: 10119–10122.

    Article  CAS  Google Scholar 

  14. Appleton AL, Curtis A, Wilkes J et al. Differentiation of materno-fetal GVHD from Omenn's syndrome in pre-BMT patients with severe combined immunodeficiency. Bone Marrow Transplant 1994; 14: 157–159.

    CAS  PubMed  Google Scholar 

  15. Barata LT, Henriques R, Hivroz C et al. Primary immunodeficiency Secondary to ZAP-70 deficiency. Acta Med Port 2001; 14: 413–417.

    CAS  PubMed  Google Scholar 

  16. Wagner JE, Kernan NA, Steinbuch M et al. Allogeneic sibling umbilical-cord-blood transplantation in children with malignant and non-malignant disease. Lancet 1995; 346: 214–219.

    Article  CAS  Google Scholar 

  17. Comans-Bitter WM, de Groot R, van den Beemd R et al. Immunophenotyping of blood lymphocytes in childhood. Reference values for lymphocyte subpopulations. J Pediatr 1997; 130: 388–393.

    Article  CAS  Google Scholar 

Download references

Acknowledgements

This work was supported by grants from the Associazione Italiana per la Ricerca sul Cancro (AIRC, Milano), from the Ministero dell'Università e della Ricerca Scientifica e Tecnologica (MURST, Roma) to E.M. and from the Associazione Donatrici Italiane di Sangue di Cordone Ombelicale (ADISCO, Sezione Piemonte).

Author information

Authors and Affiliations

  1. Department of Paediatrics, Bone Marrow Transplantation Unit, University of Turin, Italy

    F Fagioli, E Biasin, M Berger, F Nesi & E H Saroglia

  2. Department of Biological and Clinical Science, University of Turin, Italy

    R Miniero

  3. Department of Paediatrics, Infectious Diseases Unit, University of Turin, Italy

    S Martino & P A Tovo

Authors
  1. F Fagioli
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. E Biasin
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. M Berger
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. F Nesi
    View author publications

    You can also search for this author in PubMed Google Scholar

  5. E H Saroglia
    View author publications

    You can also search for this author in PubMed Google Scholar

  6. R Miniero
    View author publications

    You can also search for this author in PubMed Google Scholar

  7. S Martino
    View author publications

    You can also search for this author in PubMed Google Scholar

  8. P A Tovo
    View author publications

    You can also search for this author in PubMed Google Scholar

Rights and permissions

Reprints and permissions

About this article

Cite this article

Fagioli, F., Biasin, E., Berger, M. et al. Successful unrelated cord blood transplantation in two children with severe combined immunodeficiency syndrome. Bone Marrow Transplant 31, 133–136 (2003). https://doi.org/10.1038/sj.bmt.1703800

Download citation

  • Received:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/sj.bmt.1703800

Keywords

This article is cited by

Search

Advanced search

Quick links