Immunologic Research

, Volume 44, Issue 1, pp 25–34

Matched unrelated bone marrow transplant for Omenn syndrome

  • Amit Nahum
  • Brenda Reid
  • Eyal Grunebaum
  • Chaim M. Roifman
Article

DOI: 10.1007/s12026-008-8067-4

Cite this article as:
Nahum, A., Reid, B., Grunebaum, E. et al. Immunol Res (2009) 44: 25. doi:10.1007/s12026-008-8067-4

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.

Keywords

Omenn syndromeBone marrow transplantationMatched unrelated donorT-cell receptor excision circlesVβ families

Copyright information

© Springer Science+Business Media, LLC 2008

Authors and Affiliations

  • Amit Nahum
    • 1
    • 2
  • Brenda Reid
    • 1
    • 2
  • Eyal Grunebaum
    • 1
    • 2
  • Chaim M. Roifman
    • 1
    • 2
  1. 1.The Canadian Centre for Primary ImmunodeficiencyTorontoCanada
  2. 2.Division of Immunology and Allergy, Department of PaediatricsThe Hospital for Sick Children and The University of TorontoTorontoCanada