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 umbilical cord blood stem cell transplantation for chronic granulomatous disease

Bone Marrow Transplantation volume 31, pages 403–405 (2003)Cite this article

Summary:

Chronic granulomatous disease (CGD) causes growth failure, inflammatory lung damage and often early death. Prophylactic cotrimoxazole improves medium-term survival, but cannot prevent inflammatory sequelae. We report the first patient with CGD who underwent successful HLA identical sibling umbilical cord stem cell transplantation (UCSCT) after myeloablative conditioning. The patient presented with colitis, confirmed as CGD at 2 years of age. Following BU16/CY200 conditioning, he had UCSCT from his unaffected HLA identical sister. A year post-transplant, his colitis had resolved clinically and on radioisotope scan growth has improved. Neutrophil oxidative burst was 92% normal with full donor lymphocyte reconstitution.

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

Figure 1

Similar content being viewed by others

References

  1. Roos D, Curnutte JT . Chronic granulomatous disease. In: Ochs HD, Smith CIE, Puck JM (eds). Primary Immunodeficiency Diseases: A Molecular and Genetic Approach. Oxford University Press: Oxford, 1999, pp 353–374.

    Google Scholar 

  2. Cale CM, Jones AM, Goldblatt D . Follow up of patients with chronic granulomatous disease diagnosed since 1990. Clin Exp Immunol 2000; 120: 351–355.

    Article  CAS  Google Scholar 

  3. Gallin JI, Malech HL, Weening RS et al. A controlled trial of interferon gamma to prevent infection in chronic granulomatous disease. N Engl J Med 1991; 324: 509–516.

    Article  Google Scholar 

  4. Winkelstein JA, Marino MC, Johnston RB et al. Chronic granulomatous disease. Report on a national registry of 368 patients. Medicine 2000; 79: 155–169.

    Article  CAS  Google Scholar 

  5. Horwitz EM, Barett AJ, Brown MR et al. Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft. N Engl J Med 2001; 344: 881–888.

    Article  CAS  Google Scholar 

  6. Seger RA, Gungor T, Belohradsky BH et al. Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hematopoietic allograft: a survey of the European experience 1985–2000. Blood 2002; 100: 4344–4350.

    Article  CAS  Google Scholar 

  7. Procter SJ, Dickinson AM, Parekh T et al. Umbilical cord blood banks in the UK. BMJ 2001; 323: 60–61.

    Article  Google Scholar 

  8. Vowells SJ, Sekhsaria S, Malech HL et al. Flow cytometric analysis of the granulocyte respiratory burst: a comparison study of fluorescent probes. J Immunol Methods 1995; 178: 89–97.

    Article  CAS  Google Scholar 

  9. Gluckman E, Rocha V, Boyer-Chammand A et al. Outcome of cord-blood transplantation from related and unrelated donors. N Engl J Med 1997; 337: 373–381.

    Article  CAS  Google Scholar 

  10. 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 

  11. Dinauer MC, Gifford MA, Pech N et al. Variable correction of host defense following gene transfer and bone marrow transplantation in murine X-linked chronic granulomatous disease. Blood 2001; 97: 3738–3745.

    Article  CAS  Google Scholar 

  12. Kume A, Dinauer MC . Gene therapy for chronic granulomatous disease. J Lab Clin Med 2000; 135: 122–128.

    Article  CAS  Google Scholar 

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

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Newcastle upon Tyne Hospitals NHS Trust, University of Newcastle upon Tyne, UK

    A Bhattacharya, M Slatter, T J Flood, M Abinun, A J Cant & A R Gennery

  2. Institute of Human Genetics, University of Newcastle upon Tyne, UK

    A Curtis & A Jackson

  3. Newcastle Blood Transfusion Service, University of Newcastle upon Tyne, UK

    C E Chapman

  4. Department of Immunology, University of Newcastle upon Tyne, UK

    D Barge

Authors
  1. A Bhattacharya
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. M Slatter
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. A Curtis
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. C E Chapman
    View author publications

    You can also search for this author in PubMed Google Scholar

  5. D Barge
    View author publications

    You can also search for this author in PubMed Google Scholar

  6. A Jackson
    View author publications

    You can also search for this author in PubMed Google Scholar

  7. T J Flood
    View author publications

    You can also search for this author in PubMed Google Scholar

  8. M Abinun
    View author publications

    You can also search for this author in PubMed Google Scholar

  9. A J Cant
    View author publications

    You can also search for this author in PubMed Google Scholar

  10. A R Gennery
    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

Bhattacharya, A., Slatter, M., Curtis, A. et al. Successful umbilical cord blood stem cell transplantation for chronic granulomatous disease. Bone Marrow Transplant 31, 403–405 (2003). https://doi.org/10.1038/sj.bmt.1703863

Download citation

  • Received:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/sj.bmt.1703863

Keywords

This article is cited by

Search

Advanced search

Quick links