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.

• Författare: Nicholas Brodszki; Dominik Turkiewicz; Jacek Toporski; Lennart Truedsson; Josefina Dykes
• Avdelning/ar: Pediatrik, Lund; Avdelningen för mikrobiologi, immunologi och glykobiologi - MIG; Avdelningen för hematologi och transfusionsmedicin
• Publiceringsår: 2016
• Språk: Engelska
• Publikation/Tidskrift/Serie: Orphanet Journal of Rare Diseases
• Volym: 11
• Issue: 1
• Länkar: http://www.ncbi.nlm.nih.gov/pubmed/26768987?dopt=Abstract; http://dx.doi.org/10.1186/s13023-016-0385-3
• Dokumenttyp: Artikel i tidskrift
• Förlag: BioMed Central (BMC)
• Ämne: Pediatrics
• Status: Published
• ISBN/ISSN/Övrigt: ISSN: 1750-1172