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Conditioning Regimens

Low-intensity hematopoietic stem-cell transplantation across human leucocyte antigen barriers in dyskeratosis congenita

Bone Marrow Transplantation volume 31, pages 847–850 (2003)Cite this article

Summary:

Since the results of conventional hematopoietic stem-cell transplantation (HSCT) for patients with dyskeratosis congenita (DC) are poor owing to the high incidence of transplant-related complications, we explored the use of a low-intensity HSCT regimen. We report two children with DC with severe cytopenia, who underwent successful HSCT from a matched unrelated donor after conditioning with fludarabine, cyclophosphamide, and antithymocyte globulin. Graft-versus-host-disease (GVHD) prophylaxis consisted of corticosteroids and cyclosporin A. The regimen was well tolerated, no significant transplant-related complications were observed, and engraftment was rapid and complete. At 15 and 16 months after HSCT, the children were fully engrafted, in excellent clinical condition, full-donor chimerism, and no signs of GVHD. We conclude that a low-intensity regimen is sufficient to induce durable engraftment using matched unrelated donor HSCT in DC patients, with minimal 1-year transplant-related toxicity. Longer follow-up will determine whether this regimen also reduces long-term toxicity.

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Acknowledgements

This paper was prepared with the assistance of Editorial Services, The Hospital for Sick Children, Toronto, Ontario, Canada.

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Authors and Affiliations

  1. Marrow Failure and Myelodysplasia Programme, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada

    Y Dror & M H Freedman

  2. Division of Haematology and Oncology, Department of Paediatrics, Blood and Marrow Transplantation Section, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada

    Y Dror, M H Freedman, C A Armstrong, F E Saunders & J J Doyle

  3. London Children's Hospital, London, Ontario, Canada

    M Leaker

  4. Timmins and District Hospital, Timmins, Ontario, Canada

    J Verbeek

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Dror, Y., Freedman, M., Leaker, M. et al. Low-intensity hematopoietic stem-cell transplantation across human leucocyte antigen barriers in dyskeratosis congenita. Bone Marrow Transplant 31, 847–850 (2003). https://doi.org/10.1038/sj.bmt.1703931

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