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Matched unrelated bone marrow transplant for Omenn syndrome

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Abstract

Little information is currently available on the outcome and the long-term restoration of immune function in infants with Omenn syndrome (OS) treated with bone marrow transplantation (BMT). We prospectively followed patients with OS who received matched unrelated donor (MUD) BMT at our center. Engraftment, immune reconstitution, and transplant-related complications were recorded. Humoral and cellular immunity were evaluated. Six patients with OS were diagnosed at a mean age of 4.6 months and received a matched unrelated donor BMT as the first BMT at the mean age of 9.4 months. All six patients are alive and well at a mean 95 months after transplant. All patients have evidence of full hemopoetic engraftment and robust immune function. We have shown here that matched unrelated donor BMT is highly effective in curing patients with OS regardless of their genotype. This mode of treatment should be preferred for patients with OS when a related identical donor is not available.

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Acknowledgments

This work was supported by The Canadian Centre for Primary Immunodeficiency, The Canadian Immunodeficiency Society, and The Jeffrey Modell Foundation. CMR is holder of the Donald and Audrey Campbell Chair in Immunology.

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Authors and Affiliations

  1. The Canadian Centre for Primary Immunodeficiency, Toronto, ON, Canada

    Amit Nahum, Brenda Reid, Eyal Grunebaum & Chaim M. Roifman

  2. Division of Immunology and Allergy, Department of Paediatrics, The Hospital for Sick Children and The University of Toronto, 555 University Avenue, Toronto, ON, Canada, M5G 1X8

    Amit Nahum, Brenda Reid, Eyal Grunebaum & Chaim M. Roifman

Authors
  1. Amit Nahum
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  2. Brenda Reid
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  3. Eyal Grunebaum
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  4. Chaim M. Roifman
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Correspondence to Chaim M. Roifman.

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Nahum, A., Reid, B., Grunebaum, E. et al. Matched unrelated bone marrow transplant for Omenn syndrome. Immunol Res 44, 25–34 (2009). https://doi.org/10.1007/s12026-008-8067-4

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  • DOI: https://doi.org/10.1007/s12026-008-8067-4

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