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Challenges in the use of allogeneic hematopoietic SCT for ectodermal dysplasia with immune deficiency.

Abstract：

:Genetic mutations of proteins regulating nuclear factor of kappa-light polypeptide gene enhancer in B lymphocyte (NF-kappaB) activation result in heritable diseases of development and immunity. Hypomorphic, X-linked mutations in the IKBKG gene (NF-kappaB essential modulator (NEMO) protein), and hypermorphic, autosomal dominant mutations in the IKBA gene (inhibitor of NF-kappaB (IkappaB)-alpha protein), are associated with a phenotype of immune deficiency and often ectodermal dysplasia (ED-ID). ED-ID predisposes patients to recurrent and life-threatening infections and is typically fatal within the first few years of life. Allogeneic hematopoietic SCT (HSCT) may correct the immune deficiency associated with NEMO or IkappaBalpha mutations, but there is very little published data. We gathered clinical data on three ED-ID patients that had undergone HSCT. Conditioning regimens were variable, as were the stem cell sources. All three patients experienced engraftment difficulties as well as post transplant complications. These cases suggest that patients with immune deficiencies caused by NEMO or IkappaBalpha mutations may have intrinsic barriers to successful engraftment, which require further investigation.

journal_name

Bone Marrow Transplant

journal_title

Bone marrow transplantation

authors

Fish JD,Duerst RE,Gelfand EW,Orange JS,Bunin N

doi

10.1038/bmt.2008.308

subject

Has Abstract

pub_date

2009-02-01 00:00:00

pages

217-21

issue

3

eissn

0268-3369

issn

1476-5365

pii

bmt2008308

journal_volume

43

pub_type

杂志文章,多中心研究

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