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.

  • Correspondence
  • Published:

Allogeneic hematopoietic stem cell transplantation using unrelated cord blood or unmanipulated haploidentical donors is effective in pediatric chronic granulomatous disease with inflammatory complications and severe infection

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

Access options

Rent or buy this article

Prices vary by article type

from$1.95

to$39.95

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

Fig. 1: Outcome of allo-HSCT in pediatric chronic granulomatous disease with inflammatory complications and severe infection.

References

  1. Segal BH, Veys P, Malech H, Cowan MJ. Chronic granulomatous disease: lessons from a rare disorder. Biol Blood Marrow Transplant. 2011;17:S123–31.

    Article  CAS  Google Scholar 

  2. van den Berg JM, van Koppen E, Ahlin A, Belohradsky BH, Bernatowska E, Corbeel L, et al. Chronic granulomatous disease: the European experience. PLoS ONE. 2009;4:e5234.

    Article  Google Scholar 

  3. De Ravin SS, Naumann N, Cowen EW, Friend J, Hilligoss D, Marquesen M, et al. Chronic granulomatous disease as a risk factor for autoimmune disease. J Allergy Clin Immunol. 2008;122:1097–103.

    Article  Google Scholar 

  4. Martire B, Rondelli R, Soresina A, Pignata C, Broccoletti T, Finocchi A, et al. Clinical features, long-term follow-up and outcome of a large cohort of patients with chronic granulomatous disease: an Italian multicenter study. Clin Immunol. 2008;126:155–64.

    Article  CAS  Google Scholar 

  5. Åhlin A, Fugeläng J, de Boer M, Ringden O, Fasth A, Winiarski J. Chronic granulomatous disease-haematopoietic stem cell transplantation versus conventional treatment. Acta Paediatr. 2013;102:1087–94.

    PubMed  Google Scholar 

  6. Soncini E, Slatter MA, Jones LBKR, Hughes S, Hodges S, Flood TJ, et al. Unrelated donor and HLA-identical sibling haematopoietic stem cell transplantation cure chronic granulomatous disease with good long-term outcome and growth. Br J Haematol. 2009;45:73–83.

    Article  Google Scholar 

  7. Klein OR, Chen AR, Gamper C, Loeb D, Zambidis E, Llosa N, et al. Alternative-donor hematopoietic stem cell transplantation with post-transplantation cyclophosphamide for nonmalignant disorders. Biol Blood Marrow Transplant. 2016;22:895–901.

    Article  CAS  Google Scholar 

  8. Lum SH, Flood T, Hambleton S, McNaughton P, Watson H, Abinun M, et al. Two decades of excellent transplant survival for chronic granulomatous disease: a supraregional immunology transplant center report. Blood. 2019;23:2546–9.

    Article  Google Scholar 

  9. Nuytemans K, Vance JM. Whole exome sequencing. Rinsho Shinkeigaku. 2010;50:952–5.

    Article  Google Scholar 

  10. Ozyurek E, Cowan MJ, Koerper MA, Baxter-Lowe LA, Dvorak CC, Horn BN. Increasing mixed chimeric and the risk of graft loss in children undergoing allogeneic hematopoietic stem cell transplantation for non-malignant disorders. Bone Marrow Transplant. 2008;42:83–91.

    Article  CAS  Google Scholar 

  11. Olsson R, Remberger M, Schaffer M, Berggren DM, Svahn BM, Mattsson J, et al. Graft failure in the modern era of allogeneic hematopoietic SCT. Bone Marrow Transplant. 2013;48:537–43.

    Article  CAS  Google Scholar 

  12. Oshrine B, Morsheimer M, Heimall J, Bunin N. Reduced-intensity conditioning for hematopoietic cell transplantation of chronic granulomatous disease. Pediatr Blood Cancer. 2015;62:359–61.

    Article  Google Scholar 

  13. Parta M, Kelly C, Kwatemaa N, Theobald N, Hilligoss D, Qin J, et al. Allogeneic reduced-intensity hematopoietic stem cell transplantation for chronic granulomatous disease: a single-center prospective trial. J Clin Immunol. 2017;37:548–58.

    Article  CAS  Google Scholar 

  14. Cesaro S. Will a reduced-toxicity conditioning regimen improve the results of stem cell transplantation in metabolic disease? Bone Marrow Transplant. 2006;37:615.

    Article  CAS  Google Scholar 

  15. Khandelwal P, Bleesing JJ, Davies SM, Marsh RA. A single-center experience comparing alemtuzumab, fludarabine, and melphalan reduced-intensity conditioning with myeloablative busulfan, cyclophosphamide, and antithymocyte globulin for chronic granulomatous disease. Biol Blood Marrow Transplant. 2016;22:2011–8.

    Article  CAS  Google Scholar 

  16. Hale G, Jacobs P, Wood L, Fibbe WE, Barge R, Novitzky N, et al. CD52 antibodies for prevention of graft-versus-host disease and graft rejection following transplantation of allogeneic peripheral blood stem cells. Bone Marrow Transplant. 2000;26:69–76.

    Article  CAS  Google Scholar 

  17. 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  CAS  Google Scholar 

  18. Chang YJ, Huang XJ. Haploidentical hematopoietic stem cell transplantation with unmanipulated granulocyte colony stimulating factor mobilized marrow and blood grafts. Curr Opin Hematol. 2012;19:454–61.

    Article  CAS  Google Scholar 

  19. Gutman JA, Turtle CJ, Manley TJ, Heimfeld S, Bernstein ID, Riddell SR, et al. Single unit dominance after double-unit umbilical cord blood transplantation coincides with a specific CD8+ T-cell response against the non engrafted unit. Blood. 2010;115:757–65.

    Article  CAS  Google Scholar 

  20. Gungor T, Teira P, Slatter M, Stussi G, Stepensky P, Moshous D, et al. Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. Lancet. 2014;383:436–48.

    Article  CAS  Google Scholar 

  21. Potdar R, Varadi G, Fein J, Labopin M, Nagler A, Shouval R. Prognostic scoring systems in allogeneic hematopoietic stem cell transplantation: where do we stand? Biol Blood Marrow Transplant. 2017;23:1839–46.

    Article  Google Scholar 

  22. Törlén J, Remberge M, Le Blanc K, Ljungman P, Mattsson J. Impact of pretransplantation indices in hematopoietic stem cell transplantation: knowledge of center-specific outcome data is pivotal before making index-based decisions. Biol Blood Marrow Transplant. 2017;23:677–83.

    Article  Google Scholar 

  23. Barker JN, Wagner JE. Umbilical cord blood transplantation: current practice and future innovations. Crit Rev Oncol Hematol. 2003;48:35–43.

    Article  Google Scholar 

  24. Castillo N, García-Cadenas I, Barba P, Canals C, Díaz-Heredia C, Martino R, et al. Early and long-term impaired T lymphocyte immune reconstitution after cord blood transplantation with antithymocyte globulin. Biol Blood Marrow Transplant. 2017;23:491–7.

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Xiangfeng Tang.

Ethics declarations

Conflict of interest

The authors declare that they have no conflict of interest.

Additional information

Publisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Tang, X., Zhang, Y., Jing, Y. et al. Allogeneic hematopoietic stem cell transplantation using unrelated cord blood or unmanipulated haploidentical donors is effective in pediatric chronic granulomatous disease with inflammatory complications and severe infection. Bone Marrow Transplant 55, 1875–1878 (2020). https://doi.org/10.1038/s41409-020-0864-y

Download citation

  • Received:

  • Revised:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/s41409-020-0864-y

Search

Quick links