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Published in:

01-07-2009

Matched unrelated bone marrow transplant for Omenn syndrome

Authors: Amit Nahum, Brenda Reid, Eyal Grunebaum, Chaim M. Roifman

Published in: Immunologic Research | Issue 1-3/2009

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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Title: Matched unrelated bone marrow transplant for Omenn syndrome
Authors: Amit Nahum
Brenda Reid
Eyal Grunebaum
Chaim M. Roifman
Publication date: 01-07-2009
Publisher: Humana Press Inc
Published in: Immunologic Research / Issue 1-3/2009
Print ISSN: 0257-277X
Electronic ISSN: 1559-0755
DOI: https://doi.org/10.1007/s12026-008-8067-4

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Matched unrelated bone marrow transplant for Omenn syndrome

Abstract

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Springer Medicine

Abstract

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