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:

Matched-related allogeneic stem cell transplantation in Saudi patients with Fanconi anemia: 10 year's experience

Bone Marrow Transplantation volume 42, pages S45–S48 (2008)Cite this article

Abstract

Allogeneic SCT is curative for bone marrow failure in Fanconi anemia (FA) patients but the optimal conditioning regimen is undetermined. We report here our experience with 56 FA patients who underwent allogeneic matched related SCT. The conditioning regimen varied according to time of SCT and disease status at SCT; 22 patients (group A) received Cy 20 mg/kg, thoraco-abdominal radiation and antithymocyte globulins (ATG); and 34 patients (group B) received Cy 60 mg/kg and ATG. Median time to engraftment was similar (14 days) in both groups. Hemorrhagic cystitis was significantly more common in group B. Overall survival and event-free survival of all patients were 85 and 78.3% respectively. For groups A and B respectively, overall survival was 72.5 and 96.9% (P=0.013); and event-free survival was 72.5 and 82.3% (P=0.3). The use of the nonradiation Cy/ATG regimen in matched related SCT for FA patients offers better overall and event-free survival.

Access through your institution
Buy or subscribe

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

Access options

Access through your institution

Buy this article

  • Purchase on SpringerLink
  • Instant access to full article PDF
Buy now

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

Figure 1
Figure 2
Figure 3
Figure 4

Similar content being viewed by others

References

  1. Sieff CA, Nisbet-Brown E, Nathan D . Congenital bone marrow syndromes failures. Br J Haematol 2000; 111: 30–42.

    Article  CAS  PubMed  Google Scholar 

  2. Alter BP . Hematologic abnormalities in Fanconi anemia. Blood 1995; 85: 1148–1149.

    CAS  PubMed  Google Scholar 

  3. Auerbach AD, Weiner MA, Warburton D, Yeboa K, Lu L, Broxmeyer HE . Acute myeloid leukemia as the first hematologic manifestation of Fanconi anemia. Am J Hematol 1982; 12: 289–300.

    Article  CAS  PubMed  Google Scholar 

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

    Article  CAS  PubMed  Google Scholar 

  5. Butturini A, Gale RP, Verlander PC, Adler-Brecher B, Gillio AP, Auerbach AD . Hematologic abnormalities in Fanconi anemia: an international Fanconi anemia registry study. Blood 1994; 84: 1650–1655.

    CAS  PubMed  Google Scholar 

  6. Flowers ME, Doney KC, Storb R, Deeg HJ, Sanders JE, Sullivan KM et al. Marrow transplantation for Fanconi anemia with or without leukemic transformation: an update of the Seattle experience. Bone Marrow Transplant 1992; 9: 167–173.

    CAS  PubMed  Google Scholar 

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

    Article  CAS  PubMed  Google Scholar 

  8. Gluckman E . Allogeneic bone marrow transplantation in Fanconi anemia. Bone Marrow Transplant 1996; 18: S33–S35.

    PubMed  Google Scholar 

  9. Medeiros CR, Zanis-Neto J, Pasquini R . Bone marrow transplantation for patients with Fanconi anemia: reduced doses of cyclophosphamide without irradiation as conditioning. Bone Marrow Transplant 1999; 24: 849–852.

    Article  CAS  PubMed  Google Scholar 

  10. Gluckman E, Auerbach AD, Horowitz MM, Sobocinski KA, Ash RC, Bortin MM et al. Bone marrow transplantation for Fanconi anemia. Blood 1995; 86: 2856–2862.

    CAS  PubMed  Google Scholar 

  11. Zanis-Neto J, Flowers MED, Medeiros CR, Bitencourt MA, Bonfim CM, Setubal DC et al. Low dose cyclophosphamide conditioning for haematopoietic cell transplantation from HLA-matched related donors in patients with Fanconi anaemia. Br J Haematol 2005; 130: 99–106.

    Article  CAS  PubMed  Google Scholar 

  12. Ayas M, Al-Jefri A, Al-Mahr M, Rifai S, Moussa E, Karaoui M et al. Allogeneic stem cell transplantation in patients with Fanconi's anemia and myelodysplasia or leukemia utilizing low-dose cyclophosphamide and total body irradiation. Bone Marrow Tranpslant 2004; 33: 15–17.

    Article  CAS  Google Scholar 

  13. Dufour C, Rondelli R, Locatelli F, Miano M, Di Girolamo G, Bacigalupo A et al. Stem cell transplantation from HLA-matched related donor for Fanconi's anaemia: a retrospective review of the multicentric Italian experience on behalf of AIEOP-GITMO. Br J Haematol 2001; 112: 796–805.

    Article  CAS  PubMed  Google Scholar 

  14. Ayas M, Al-Jefri A, Al-Mahr M, Rifai S, Al-Seraihi A, Tbakhi A et al. Stem cell transplantation for patients with Fanconi anemia with low-dose cyclophosphamide and antithymocyte globulins without the use of radiation therapy. Bone Marrow Tranpslant 2005; 35: 463–466.

    Article  CAS  Google Scholar 

  15. Socié G, Devergie A, Girinski T, Piel G, Ribaud P, Esperou H 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  PubMed  Google Scholar 

  16. Sullivan KM . Graft-versus-host disease. In: Forman SJ, Blume KG, Thomas ED (eds). Bone Marrow Transplantation. Blackwell Scientific Publications: Boston, 1997, pp 339.

    Google Scholar 

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

    Article  CAS  PubMed  Google Scholar 

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

    Article  CAS  PubMed  Google Scholar 

  19. Kohli-Kumar M, Morris C, DeLaat C, Sambrano J, Masterson M, Mueller R et al. Bone marrow transplantation in Fanconi anemia using matched sibling donors. Blood 1994; 84: 2050–2054.

    CAS  PubMed  Google Scholar 

  20. Ayas M, Solh H, Mustafa MM, Al-Mahr M, Al-Fawaz I, Al-Jefri A et al. Bone marrow transplantation from matched siblings in patients with Fanconi anemia utilizing low-dose cyclophosphamide, thoracoabdominal radiation and antithymocyte globulin. Bone Marrow Transplant 2001; 27: 139–143.

    Article  CAS  PubMed  Google Scholar 

  21. Deeg HJ, Socie G, Schoch G, Henry-Amar M, Witherspoon RP, Devergie A et al. Malignancies after marrow transplantation for aplastic and Fanconi anemia: a joint Seattle and Paris analysis of results in 700 patients. Blood 1996; 87: 386–392.

    CAS  PubMed  Google Scholar 

  22. Kutler DI, Singh B, Satagopan J, Batish SD, Berwick M, Giampietro PF et al. A 20-year perspective on the international Fanconi anemia registry (IFAR). Blood 2003; 101: 1249–1256.

    Article  CAS  PubMed  Google Scholar 

  23. Kutler DI, Auerbach AD, Satagopan J, Giampietro PF, Batish SD, Huvos AG et al. High incidence of head and neck squamous cell carcinoma in patients with Fanconi anemia. Arch Otolaryngol 2003; 129: 106–112.

    Article  Google Scholar 

  24. Tan PL, Wagner JE, Auerbach AD, Defor TE, Slungaard A, Macmillan ML . Successful engraftment without radiation after fludarabine-based regimen in Fanconi anemia patients undergoing genotypically identical donor hematopoietic cell transplantation. Pediatr Blood Cancer 2006; 46: 630–636.

    Article  PubMed  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Department of Pediatric Hematology/Oncology, The Section of Pediatric Stem Cell Transplant, Riyadh, Saudi Arabia

    M Ayas, A Al-Jefri, A Al-Seraihi, M Al-Mahr & H El-Solh

  2. The Department of Biostatistics and Epidemiology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia

    N Elkum

Authors
  1. M Ayas
    View author publications

    Search author on:PubMed Google Scholar

  2. A Al-Jefri
    View author publications

    Search author on:PubMed Google Scholar

  3. A Al-Seraihi
    View author publications

    Search author on:PubMed Google Scholar

  4. N Elkum
    View author publications

    Search author on:PubMed Google Scholar

  5. M Al-Mahr
    View author publications

    Search author on:PubMed Google Scholar

  6. H El-Solh
    View author publications

    Search author on:PubMed Google Scholar

Corresponding author

Correspondence to M Ayas.

Rights and permissions

Reprints and permissions

About this article

Cite this article

Ayas, M., Al-Jefri, A., Al-Seraihi, A. et al. Matched-related allogeneic stem cell transplantation in Saudi patients with Fanconi anemia: 10 year's experience. Bone Marrow Transplant 42 (Suppl 1), S45–S48 (2008). https://doi.org/10.1038/bmt.2008.114

Download citation

  • Published:

  • Issue date:

  • DOI: https://doi.org/10.1038/bmt.2008.114

Keywords

This article is cited by

Search

Advanced search

Quick links