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Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency

Lookup NU author(s): Professor Mary Slatter, Dr Terence Flood, Dr Mario Abinun, Professor Andrew Cant, Professor Andrew GenneryORCiD


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Primary immunodeficiencies (PID) are an important cause of childhood mortality. Haematopoietic stem cell transplantation (HSCT) is the best treatment for many PID. Umbilical cord stem cells are an alternative source of HSC. There is little data regarding outcome of umbilical cord stem cell transplantation (UCSCT) for PID. Our single centre experience is reported. A retrospective study of 14 of 148 patients transplanted for PID, who have received 15 UCSCT was performed, with specific regard to graft-versus-host disease (GvHD) and immune reconstitution. Eight patients with severe combined immunodeficiency (SCID), and six with other combined immunodeficiencies were treated. Of the patients, 12 received unrelated cords, and two had sibling transplants. Median age at transplant was 3.5 months, median nucleated cell dose was 0.8 × 108/kg. All engrafted. Median time to neutrophil engraftment was 22 days, median time to platelet engraftment was 51 days. One developed significant grade III GvHD post transplantation. In total, 11 patients had full donor T and six full donor B-cell chimerism, six of nine patients > 1 year post-BMT had normal IgG levels and specific antibody responses to tetanus and Hib vaccines; two are being assessed. Two patients died of multi-organ failure related to pre-existing infection and inflammatory complications respectively. UCSCT should be considered for patients requiring stem cell therapy for PID. © 2005 Nature Publishing Group. All rights reserved.

Publication metadata

Author(s): Bhattacharya A, Slatter MA, Chapman CE, Barge D, Jackson A, Flood TJ, Abinun M, Cant AJ, Gennery AR

Publication type: Article

Publication status: Published

Journal: Bone Marrow Transplantation

Year: 2005

Volume: 36

Issue: 4

Pages: 295-299

Print publication date: 01/08/2005

ISSN (print): 0268-3369

ISSN (electronic): 1476-5365

Publisher: Nature Publishing Group


DOI: 10.1038/sj.bmt.1705054

PubMed id: 15968287


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