Skip to main content

Advertisement

Log in

Successful allogeneic hematopoietic stem cell transplantation for chronic granulomatous disease with inflammatory complications and severe infection

  • Case Report
  • Published:
International Journal of Hematology Aims and scope Submit manuscript

Abstract

We report two patients with chronic granulomatous disease (CGD). The first patient presented with granulomatous colitis and pulmonary aspergillosis, and the second patient with liver abscess and restrictive pulmonary disorder. Both patients underwent allogeneic hematopoietic stem cell transplantation, the first from an HLA-matched sibling donor, and the second from an HLA-matched unrelated donor, after preconditioning with fludarabine, anti-thymocyte globulin, cyclophosphamide, and total-body irradiation of 3 Gy. The engraftment was prompt and the regimen-related toxicity was mild. The patients are able to return to their daily lives with full donor chimerism, although the second patient underwent a living-related-donor orthotopic liver transplantation from his mother for chronic liver graft-versus-host disease. The conditioning regimen we used was feasible and applicable to patients with CGD accompanied by inflammatory disease and severe infection.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

Similar content being viewed by others

References

  1. Dinauer MC. Chronic granulomatous disease and other disorders of phagocyte function. Hematology 2005:89–95.

  2. Seger RA, Gungor T, Belohradsky BH, Blanche S, Bordigoni P, et al. Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985–2000. Blood. 2002;100:4344–50.

    Article  PubMed  CAS  Google Scholar 

  3. Horwitz ME, Barrett AJ, Brown MR, Carter CS, Childs R, et al. Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft. N Engl J Med. 2001;344:881–8.

    Article  PubMed  CAS  Google Scholar 

  4. Yokoyama S, Kasahara M, Fukuda A, Sato S, Mori T, Nakagawa A, Matsui A. Successful living-donor liver transplantation for chronic hepatic graft-versus-host disease after bone marrow transplantation for chronic granulomatous disease. Transplantation. 2008;86:367–8.

    Article  PubMed  Google Scholar 

  5. Ott MG, Schmidt M, Schwarzwaelder K, et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med. 2006;12:401–9.

    Article  PubMed  CAS  Google Scholar 

  6. Parikh SH, Szabolcs P, Prasad VK, Lakshminarayanan S, Martin PL, Driscoll TA, Kurtzberg J. Correction of chronic granulomatous disease after second unrelated-donor umbilical cord blood transplantation. Pediatr Blood Cancer. 2007;49:982–4.

    Article  PubMed  Google Scholar 

  7. Nicholson JA, Wynn RF, Carr TF, Will AM. Sequential reduced- and full-intensity allografting using same donor in a child with chronic granulomatous disease and coexistent, significant comorbidity. Bone Marrow Transplant. 2004;34:1009–10.

    Article  PubMed  CAS  Google Scholar 

  8. Del Giudice I, Iori AP, Mengarelli A, et al. Allogeneic stem cell transplant from HLA-identical sibling for chronic granulomatous disease and review of the literature. Ann Hematol. 2003;82:189–92.

    PubMed  CAS  Google Scholar 

  9. Ljungman P, Hassan M, Békássy AN, Ringdén O, Oberg G. Busulfan concentration in relation to permanent alopecia in recipients of bone marrow transplants. Bone Marrow Transplant. 1995;15:869–71.

    PubMed  CAS  Google Scholar 

  10. Locatelli F, Giorgiani G, Pession A, Bozzola M. Late effects in children after bone marrow transplantation: a review. Haematologica. 1993;78:319–28.

    PubMed  CAS  Google Scholar 

  11. Ringdén O, Remberger M, Ruutu T, et al. Increased risk of chronic graft-versus-host disease, obstructive bronchiolitis, and alopecia with busulfan versus total body irradiation: long-term results of a randomized trial in allogeneic marrow recipients with leukemia. Nordic Bone Marrow Transplantation Group. Blood. 1999;93:2196–201.

    PubMed  Google Scholar 

  12. Clift RA, Radich J, Appelbaum FR, et al. Long-term follow-up of a randomized study comparing cyclophosphamide and total body irradiation with busulfan and cyclophosphamide for patients receiving allogenic marrow transplants during chronic phase of chronic myeloid leukemia. Blood. 1999;94:3960–2.

    PubMed  CAS  Google Scholar 

  13. Akioka S, Itoh H, Ueda I, et al. Donor lymphocyte infusion at unstable mixed chimerism in an allogeneic BMT recipient for chronic granulomatous disease. Bone Marrow Transplant. 1998;22:609–11.

    Article  PubMed  CAS  Google Scholar 

  14. Güngör T, Halter J, Klink A, Junge S, Stumpe KD, Seger R, Schanz U. Successful low toxicity hematopoietic stem cell transplantation for high-risk adult chronic granulomatous disease patients. Transplantation. 2005;79:1596–606.

    Article  PubMed  Google Scholar 

  15. Bacigalupo A, Ballen K, Rizzo D, et al. Defining the intensity of conditioning regimens: working definitions. Biol Blood Marrow Transplant. 2009;15:1628–33.

    Article  PubMed  Google Scholar 

  16. Nagler A, Ackerstein A, Kapelushnik J, Or R, Naparstek E, Slavin S. Donor lymphocyte infusion post-non-myeloablative allogeneic peripheral blood stem cell transplantation for chronic granulomatous disease. Bone Marrow Transplant. 1999;24:339–42.

    Article  PubMed  CAS  Google Scholar 

  17. Sastry J, Kakakios A, Tugwell H, Shaw PJ. Allogeneic bone marrow transplantation with reduced intensity conditioning for chronic granulomatous disease complicated by invasive Aspergillus infection. Pediatr Blood Cancer. 2006;47:327–9.

    Article  PubMed  Google Scholar 

  18. Hara K, Kajiume T, Kondo T, Sera Y, Kawaguchi H, Kobayashi M. Respiratory complications after haematopoietic stem cell transplantation in a patient with chronic granulomatous disease. Transfus Med. 2009;19:105–8.

    Article  PubMed  CAS  Google Scholar 

Download references

Acknowledgments

We must clarify M.T. is a recipient of Grant for Research on Allergic disease and Immunology (H20-016) from Japanese Ministry of Health, Labor and Welfare.

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Keisuke Kato.

Additional information

K. Kato and Y. Kojima are contributed equally to this work.

About this article

Cite this article

Kato, K., Kojima, Y., Kobayashi, C. et al. Successful allogeneic hematopoietic stem cell transplantation for chronic granulomatous disease with inflammatory complications and severe infection. Int J Hematol 94, 479–482 (2011). https://doi.org/10.1007/s12185-011-0932-6

Download citation

  • Received:

  • Revised:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/s12185-011-0932-6

Keywords

Navigation