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Successful umbilical cord blood stem cell transplantation for chronic granulomatous disease

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


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Chronic granulomatous disease (CGD) causes growth failure, inflammatory lung damage and often early death. Prophylactic cotrimoxazole improves medium-term survival, but cannot prevent inflammatory sequelae. We report the first patient with CGD who underwent successful HLA identical sibling umbilical cord stem cell transplantation (UCSCT) after myeloablative conditioning. The patient presented with colitis, confirmed as CGD at 2 years of age. Following BU16/CY200 conditioning, he had UCSCT from his unaffected HLA identical sister. A year post-transplant, his colitis had resolved clinically and on radioisotope scan growth has improved. Neutrophil oxidative burst was 92% normal with full donor lymphocyte reconstitution.

Publication metadata

Author(s): Bhattacharya A, Slatter M, Curtis A, 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: 2003

Volume: 31

Issue: 5

Pages: 403-405

Print publication date: 01/03/2003

ISSN (print): 0268-3369

ISSN (electronic): 1476-5365

Publisher: Nature Publishing Group


DOI: 10.1038/sj.bmt.1703863

PubMed id: 12634733


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