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.

  • Review
  • Published:

HSCT for Fanconi anemia in children: factors that influence early and late results

Bone Marrow Transplantation volume 42, pages S51–S53 (2008)Cite this article

Abstract

Fanconi anemia (FA) is a rare autosomal recessive disease characterized by congenital abnormalities, cancer predisposition and progressive BM failure. FA patients present spontaneous and induced chromosome breakage. Hematopoietic SCT (HSCT) represents the unique therapeutic option to restore normal hematopoiesis when marrow failure or clonal hematopoietic abnormality occurs. Conventional myeloablative conditioning regimen, especially including a high dose of irradiation, appeared strongly toxic for FA patients. Then, reduced-intensity conditioning regimens were developed successfully for those patients. However, TRM still remained higher than for other HSCT indications. The development of fludarabine containing a non-myeloablative conditioning regimen appears to be a major progress. Long-term follow-up is absolutely necessary.

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. D’Andrea AD, Dahl N, Guinan EC, Shimamura A . Marrow failure. Hematology 2002; 58–72.

  2. Bagby GC, Alter BP . Fanconi anemia. Semin Hematol 2006; 43: 147–156.

    Article  CAS  Google Scholar 

  3. Mace G, Briot D, Guervilly JH, Rosselli F . Fanconi anemia: cellular and molecular features. Pathol Biol (Paris) 2007; 55: 19–28.

    Article  CAS  Google Scholar 

  4. Soulier J, Leblanc T, Larghero J, Dastot H, Shimamura A, Guardiola P et al. Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 2005; 105: 1329–1336.

    Article  CAS  Google Scholar 

  5. van Zeeburg HJ, Snijders PJ, Pals G, Hermsen MA, Rooimans MA, Bagby G et al. Generation and molecular characterization of head and neck squamous cell lines of fanconi anemia patients. Cancer Res 2005; 65: 1271–1276.

    Article  CAS  Google Scholar 

  6. Alter BP, Greene MH, Velazquez I, Rosenberg PS . Cancer in Fanconi anemia. Blood 2003; 101: 2072.

    Article  CAS  Google Scholar 

  7. Rosenberg PS, Alter BP, Ebell W . Cancer risks in Fanconi anemia: findings from the German Fanconi Anemia Registry. Haematologica 2008; 93: 511–517.

    Article  Google Scholar 

  8. Rosenberg PS, Greene MH, Alter BP . Cancer incidence in persons with Fanconi anemia. Blood 2003; 101: 822–826.

    Article  CAS  Google Scholar 

  9. Berger R, Bernheim A, Gluckman E, Gisselbrecht C . In vitro effect of cyclophosphamide metabolites on chromosomes of Fanconi anaemia patients. Br J Haematol 1980; 45: 565–568.

    Article  CAS  Google Scholar 

  10. Gluckman E, Devergie A, Dutreix J . Radiosensitivity in Fanconi anaemia: application to the conditioning regimen for bone marrow transplantation. Br J Haematol 1983; 54: 431–440.

    Article  CAS  Google Scholar 

  11. Gluckman E, Devergie A, Schaison G, Bussel A, Berger R, Sohier J et al. Bone marrow transplantation in Fanconi anaemia. Br J Haematol 1980; 45: 557–564.

    Article  CAS  Google Scholar 

  12. Socie G, Devergie A, Girinski T, Piel G, Ribaud P, Esperou H, Parquet N et al. Transplantation for Fanconi's anaemia: long-term follow-up of fifty patients transplanted from a sibling donor after low-dose cyclophosphamide and thoraco-abdominal irradiation for conditioning. Br J Haematol 1998; 103: 249–255.

    Article  CAS  Google Scholar 

  13. Flowers ME, Zanis J, Pasquini R, Deeg HJ, Ribeiro R, Longton G et al. Marrow transplantation for Fanconi anaemia: conditioning with reduced doses of cyclophosphamide without radiation. Br J Haematol 1996; 92: 699–706.

    Article  CAS  Google Scholar 

  14. Gluckman E, Wagner JE . Hematopoietic stem cell transplantation in childhood inherited bone marrow failure syndrome. Bone Marrow Transplant 2008; 41: 127–132.

    Article  CAS  Google Scholar 

  15. Kapelushnik J, Or R, Slavin S, Nagler A . A fludarabine-based protocol for bone marrow transplantation in Fanconi's anemia. Bone Marrow Transplant 1997; 20: 1109–1110.

    Article  CAS  Google Scholar 

  16. Wagner JE, Eapen M, MacMillan ML, Harris RE, Pasquini R, Boulad F et al. Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia. Blood 2007; 109: 2256–2262.

    Article  CAS  Google Scholar 

  17. Yabe M, Yabe H, Hamanoue S, Inoue H, Matsumoto M, Koike T et al. In vitro effect of fludarabine, cyclophosphamide, and cytosine arabinoside on chromosome breakage in Fanconi anemia patients: relevance to stem cell transplantation. Int J Hematol 2007; 85: 354–361.

    Article  CAS  Google Scholar 

  18. Farzin A, Davies SM, Smith FO, Filipovich A, Hansen M, Auerbach AD et al. Matched sibling donor haematopoietic stem cell transplantation in Fanconi anaemia: an update of the Cincinnati Children's experience. Br J Haematol 2007; 136: 633–640.

    Article  CAS  Google Scholar 

  19. Ayas M, Al-Jefri A, Al-Seraihi A, Al-Mahr M, Rifai S, Al-Ahmari A et al. Allogeneic stem cell transplantation in Fanconi anemia patients presenting with myelodysplasia and/or clonal abnormality: update on the Saudi experience. Bone Marrow Transplant 2008; 41: 261–265.

    Article  CAS  Google Scholar 

  20. Bitan M, Or R, Shapira MY, Aker M, Resnick IB, Ackerstein A et al. Fludarabine-based reduced intensity conditioning for stem cell transplantation of Fanconi anemia patients from fully matched related and unrelated donors. Biol Blood Marrow Transplant 2006; 12: 712–718.

    Article  CAS  Google Scholar 

  21. Locatelli F, Zecca M, Pession A, Morreale G, Longoni D, Di Bartolomeo P et al. The outcome of children with Fanconi anemia given hematopoietic stem cell transplantation and the influence of fludarabine in the conditioning regimen: a report from the Italian pediatric group. Haematologica 2007; 92: 1381–1388.

    Article  Google Scholar 

  22. Gluckman E, Rocha V, Ionescu I, Bierings M, Harris RE, Wagner J et al. Results of unrelated cord blood transplant in fanconi anemia patients: risk factor analysis for engraftment and survival. Biol Blood Marrow Transplant 2007; 13: 1073–1082.

    Article  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Pediatric Immuno-Hematology Unit, Robert Debré Hospital, Paris, France

    J-H Dalle

  2. Paris-Diderot, Paris-7 University, Paris, France

    J-H Dalle

Authors
  1. J-H Dalle
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to J-H Dalle.

Rights and permissions

Reprints and permissions

About this article

Cite this article

Dalle, JH. HSCT for Fanconi anemia in children: factors that influence early and late results. Bone Marrow Transplant 42 (Suppl 2), S51–S53 (2008). https://doi.org/10.1038/bmt.2008.284

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/bmt.2008.284

Keywords

This article is cited by

Search

Advanced search

Quick links