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Hematopoietic stem cell transplantation for primary immunodeficiency diseases

Lookup NU author(s): Professor Mary Slatter, Professor Andrew Cant


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Hematopoietic stem cell transplantation (HSCT) is now highly successfully curing a widening range of primary immunodeficiencies (PIDs). Better tissue typing, matching of donors, less toxic chemotherapy, better virus detection and treatment, improved supportive care, and graft-versus-host disease prophylaxis mean up to a 90% cure for severe combined immunodeficiency patients and a 70-80% cure for other PIDs given a matched unrelated donor, and rising to 95% for young patients with specific PIDs, such as Wiskott-Aldrich syndrome. Precise molecular diagnosis, detailed data on prognosis, and careful pre-HSCT assessment of infective lung and liver damage will ensure an informed benefit analysis of HSCT and the best outcome. It is now recognized that the best treatment option for chronic granulomatous disease is HSCT, which can also be curative for CD40 ligand deficiency and complex immune dysregulation disorders.

Publication metadata

Author(s): Slatter MA, Cant AJ

Editor(s): Casanova, J.L., Conley, M.E., Notarengelo, L.

Publication type: Book Chapter

Publication status: Published

Book Title: The Year in Human and Medical Genetics: Inborn Errors of Immunity I

Year: 2011

Volume: 1238

Pages: 122-131

Print publication date: 01/01/2011

Series Title: Annals of the New York Academy of Sciences

Publisher: Wiley-Blackwell

Place Published: Oxford, UK


DOI: 10.1111/j.1749-6632.2011.06243.x

Notes: ISSN: 0077-8923 (print) 1749-6632 (online)

Library holdings: Search Newcastle University Library for this item

ISBN: 9781573318495