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

Research output: Contribution to journalArticle

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.

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Subject classification (UKÄ) – MANDATORY

  • Pediatrics
Original languageEnglish
Article number5
JournalOrphanet Journal of Rare Diseases
Volume11
Issue number1
Publication statusPublished - 2016
Publication categoryResearch
Peer-reviewedYes

Related research output

Brodszki, N., 2016, Paediatrics, Faculty of Medicine, Lund University. 148 p.

Research output: ThesisDoctoral Thesis (monograph)

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