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

Summary, in English

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: Nicholas Brodszki; Dominik Turkiewicz; Jacek Toporski; Lennart Truedsson; Josefina Dykes
• Department/s: Paediatrics (Lund); Division of Microbiology, Immunology and Glycobiology - MIG; Division of Hematology and Transfusion Medicine
• Publishing year: 2016
• Language: English
• Publication/Series: Orphanet Journal of Rare Diseases
• Volume: 11
• Issue: 1
• Links: http://www.ncbi.nlm.nih.gov/pubmed/26768987?dopt=Abstract; http://dx.doi.org/10.1186/s13023-016-0385-3
• Document type: Journal article
• Publisher: BioMed Central (BMC)
• Topic: Pediatrics
• Status: Published
• ISBN/ISSN/Other: ISSN: 1750-1172