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Fanconi Anemia

Bone marrow transplantation from matched siblings in patients with Fanconi anemia utilizing low-dose cyclophosphamide, thoracoabdominal radiation and antithymocyte globulin

Bone Marrow Transplantation volume 27, pages 139–143 (2001)Cite this article

Abstract

Nineteen patients with Fanconi anemia (FA) and bone marrow failure underwent bone marrow transplantation (BMT) from matched siblings. Median age at BMT was 8.7 years. Conditioning consisted of low-dose cyclophosphamide (CY 5 mg/kg × 4 days) and thoracoabdominal irradiation (TAI 400 cGy). Graft-versus-host disease (GVHD) prophylaxis was cyclosporin A (CsA) in 13 patients and CsA plus methotrexate in 6 patients. Antithymocyte globulin (ATG) was added in the pretransplant as well as the post-transplant period. All patients received high-dose acyclovir from day 2 pre-BMT to day 28 post BMT, and intravenous immunoglobulins (IVIG), 500 mg/kg weekly from day 7 pre-BMT to day 90 post BMT. No fungal prophylaxis was given. All patients engrafted, (median, 14 days for an absolute neutrophil count ⩾0.5 × 109/l; median, 37 days for platelet count ⩾20 × 109/l). Fourteen (74%) patients are alive with sustained engraftment and are transfusion independent. Three (16.6%) patients developed acute GVHD; none developed chronic GVHD. Five (26%) patients developed invasive fungal infections, and two (10%) developed fatal CMV disease. We believe the addition of ATG may have contributed to the increased incidence of severe life-threatening fungal and viral infections in our series. Bone Marrow Transplantation (2001) 27, 139–143.

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Acknowledgements

We are extremely grateful to Dr Robert Stuart, (chairman of the Department of Oncology at the King Faisal Specialist Hospital) for his critical review of this manuscript. We are also grateful to Mrs Saada Mansour (clinical research coordinator) and to Mrs Cynthia Magpantay (research data analyst) for their excellent assistance in the data collection and analysis.

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  1. Department of Oncology, King Faisal Specialist Hospital and Research Center (KFSH&RC), Riyadh, Saudi Arabia

    M Ayas, H Solh, MM Mustafa, M Al-Mahr, I Al-Fawaz, A Al-Jefri, L Shalaby, A Al-Nasser & R Al-Sedairy

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  1. M Ayas
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Ayas, M., Solh, H., Mustafa, M. 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 27, 139–143 (2001). https://doi.org/10.1038/sj.bmt.1702754

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