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Allografting

Stem cell transplantation for patients with Fanconi anemia with low-dose cyclophosphamide and antithymocyte globulins without the use of radiation therapy

Bone Marrow Transplantation volume 35, pages 463–466 (2005)Cite this article

Summary:

In all, 22 patients with confirmed Fanconi anemia (FA) underwent stem cell transplantation (SCT) from HLA-matched, related donors at KFSHRC. Median age at SCT was 7.6 years (range, 2.5–14.6 years). Conditioning regimen consisted of cyclophosphamide (CY) 15 mg/kg/day intravenously (i.v.) for 4 consecutive days, in addition to equine antithymocyte globulins (ATG) given i.v. at 40 mg/kg/day for four doses pre-SCT. No radiation therapy was given. For graft-versus-host disease prophylaxis, we used cyclosporin at the standard doses; ATG was added at 20 mg/kg/dose i.v. on days 2, 4, 6, 8, 10, and 12 post-SCT (total of six doses). All patients engrafted and are alive and transfusion independent with a median follow-up time of 20.2 months (range, 3.3–59 months). One patient however developed a decrease in her WBC and platelet count. Her work-up revealed slightly hypocellular bone marrow, and a series of chimerism studies over 1 year confirmed that she has stable mixed chimerism; she remains transfusion independent. We conclude that low-dose CY without radiation therapy can be used satisfactorily in the conditioning of patients with FA undergoing related SCT.

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Authors and Affiliations

  1. Department of Pediatric Hematology-Oncology, King Faisal Specialist Hospital and Research Center (KFSHRC), Riyadh, Saudi Arabia

    M Ayas, A Al-Jefri, M Al-Mahr, S Rifai, A Al-Seraihi, A Khairy, E Moussa, L Shalaby & H El-Solh

  2. Department of Pathology and Laboratory Medicine, KFSHRC, Riyadh, Saudi Arabia

    A Tbakhi & A Iqbal

  3. Department of Pediatrics, King Hussein Cancer Center, Amman, Jordan

    M Mustafa

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  1. M Ayas
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  2. A Al-Jefri
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  3. M Al-Mahr
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  4. S Rifai
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  5. A Al-Seraihi
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  7. M Mustafa
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  11. L Shalaby
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  12. H El-Solh
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Correspondence to M Ayas.

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Ayas, M., Al-Jefri, A., Al-Mahr, M. 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 Transplant 35, 463–466 (2005). https://doi.org/10.1038/sj.bmt.1704787

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