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Novel treatment of severe combined immunodeficiency utilizing ex-vivo T-cell depleted haploidentical hematopoietic stem cell transplantation and CD45RA+ depleted donor lymphocyte infusions.

Brodszki, Nicholas LU ; Turkiewicz, Dominik ; Toporski, Jacek LU ; Truedsson, Lennart LU and Dykes, Josefina LU (2016) In Orphanet Journal of Rare Diseases 11(1).
Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment available for severe combined immunodeficiency (SCID); although, there is a high incidence of severe infections and an increased risk of graft-versus host-disease (GvHD) with HSCT. Early intervention is a crucial prognostic factor and a HLA-haploidentical parental donor is often available. Haploidentical HSCT protocols utilizing extensively ex vivo T-cell depleted grafts (CliniMACs system) have proven efficient in preventing GvHD, but cause a delay in early T-cell recovery that increases the risk of viral infections. Here, we present a novel approach for treating SCID that combines selective depletion of GvHD-inducing alpha/beta (α/β) T-cells from the... (More)
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment available for severe combined immunodeficiency (SCID); although, there is a high incidence of severe infections and an increased risk of graft-versus host-disease (GvHD) with HSCT. Early intervention is a crucial prognostic factor and a HLA-haploidentical parental donor is often available. Haploidentical HSCT protocols utilizing extensively ex vivo T-cell depleted grafts (CliniMACs system) have proven efficient in preventing GvHD, but cause a delay in early T-cell recovery that increases the risk of viral infections. Here, we present a novel approach for treating SCID that combines selective depletion of GvHD-inducing alpha/beta (α/β) T-cells from the haploidentical HSCT graft with a subsequent donor lymphocyte infusion (DLI) enriched for CD45RO+ memory T-cells. (Less)
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author
; ; ; and
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Orphanet Journal of Rare Diseases
volume
11
issue
1
article number
5
publisher
BioMed Central (BMC)
external identifiers
  • pmid:26768987
  • wos:000368141100001
  • scopus:84954088086
  • pmid:26768987
ISSN
1750-1172
DOI
10.1186/s13023-016-0385-3
language
English
LU publication?
yes
id
6722c947-9908-40b7-9f1d-0ab472aadb16 (old id 8589110)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/26768987?dopt=Abstract
date added to LUP
2016-04-01 13:25:20
date last changed
2024-10-10 02:18:20
@article{6722c947-9908-40b7-9f1d-0ab472aadb16,
  abstract     = {{Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment available for severe combined immunodeficiency (SCID); although, there is a high incidence of severe infections and an increased risk of graft-versus host-disease (GvHD) with HSCT. Early intervention is a crucial prognostic factor and a HLA-haploidentical parental donor is often available. Haploidentical HSCT protocols utilizing extensively ex vivo T-cell depleted grafts (CliniMACs system) have proven efficient in preventing GvHD, but cause a delay in early T-cell recovery that increases the risk of viral infections. Here, we present a novel approach for treating SCID that combines selective depletion of GvHD-inducing alpha/beta (α/β) T-cells from the haploidentical HSCT graft with a subsequent donor lymphocyte infusion (DLI) enriched for CD45RO+ memory T-cells.}},
  author       = {{Brodszki, Nicholas and Turkiewicz, Dominik and Toporski, Jacek and Truedsson, Lennart and Dykes, Josefina}},
  issn         = {{1750-1172}},
  language     = {{eng}},
  number       = {{1}},
  publisher    = {{BioMed Central (BMC)}},
  series       = {{Orphanet Journal of Rare Diseases}},
  title        = {{Novel treatment of severe combined immunodeficiency utilizing ex-vivo T-cell depleted haploidentical hematopoietic stem cell transplantation and CD45RA+ depleted donor lymphocyte infusions.}},
  url          = {{http://dx.doi.org/10.1186/s13023-016-0385-3}},
  doi          = {{10.1186/s13023-016-0385-3}},
  volume       = {{11}},
  year         = {{2016}},
}