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Fanconi Anaemia

Non-TBI stem cell transplantation protocol for Fanconi anaemia using HLA-compatible sibling and unrelated donors

Bone Marrow Transplantation volume 32, pages 653–656 (2003)Cite this article

A Corrigendum to this article was published on 21 June 2004

Summary:

Allogeneic haemopoietic stem cell transplantation (SCT) is the only curative option for severe bone marrow (BM) failure in patients with Fanconi anaemia (FA). We have developed a non total body irradiation (TBI) conditioning protocol consisting of fludarabine (120–150 mg/m2), low dose of cyclophosphamide (40 mg/kg) and antilymphocyte globulin (45 mg/kg). Graft-versus-host disease (GVHD) prophylaxis was with cyclosporin alone for sibling allografts but also included Campath-1 H (days 1–5 post SCT) for the unrelated allografts. We have performed two sibling and two unrelated BM transplants with a follow-up of 11–51 months. All patients experienced minimal toxicity and were discharged from hospital 28–32 days post SCT. Neutrophil and platelet engraftment occurred from days 11 to 19 and 15 to 34, respectively. All patients achieved stable full donor haemopoiesis with normalisation of the peripheral blood count despite one of them having myelodysplasia (MDS) with 8% blasts prior to the SCT. The only site of acute GVHD was in the skin (grade I–II) and only one patient progressed to limited chronic GVHD. This protocol is associated with reduced toxicity and prompt engraftment in FA patients with AA and/or MDS undergoing SCT using sibling or unrelated donors.

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Acknowledgements

We are grateful to all the medical and nursing staff involved in the management of the patients in this study.

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Authors and Affiliations

  1. Department of Haematology, Imperial College of Science, Technology and Medicine, London, UK

    J de la Fuente, T Vulliamy, I A G Roberts, A Rahemtulla & I Dokal

  2. Department of Haematology, Hammersmith Hospital, London, UK

    J de la Fuente, S Reiss, T Vulliamy, A Rahemtulla & I Dokal

  3. Department of Paediatrics, St Mary's Hospital, London, UK

    M McCloy & I A G Roberts

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  1. J de la Fuente
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  2. S Reiss
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  3. M McCloy
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  4. T Vulliamy
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  6. A Rahemtulla
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  7. I Dokal
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Correspondence to J de la Fuente.

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Fuente, J., Reiss, S., McCloy, M. et al. Non-TBI stem cell transplantation protocol for Fanconi anaemia using HLA-compatible sibling and unrelated donors. Bone Marrow Transplant 32, 653–656 (2003). https://doi.org/10.1038/sj.bmt.1704219

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