Successful umbilical cord blood stem cell transplantation for chronic granulomatous disease

被引:29
作者
Bhattacharya, A
Slatter, M
Curtis, A
Chapman, CE
Barge, D
Jackson, A
Flood, TJ
Abinun, M
Cant, AJ
Gennery, AR
机构
[1] Univ Newcastle Upon Tyne, Dept Immunol, Newcastle Upon Tyne NE1 7RU, Tyne & Wear, England
[2] Univ Newcastle Upon Tyne, Newcastle Blood Transfus Serv, Newcastle Upon Tyne NE1 7RU, Tyne & Wear, England
[3] Univ Newcastle Upon Tyne, Inst Human Genet, Newcastle Upon Tyne NE1 7RU, Tyne & Wear, England
[4] Univ Newcastle Upon Tyne, Newcastle Upon Tyne Hosp NHS Trust, Newcastle Upon Tyne NE1 7RU, Tyne & Wear, England
关键词
chronic granulomatous disease; umbilical cord blood; allogencic transplantation;
D O I
10.1038/sj.bmt.1703863
中图分类号
Q6 [生物物理学];
学科分类号
071011 ;
摘要
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.
引用
收藏
页码:403 / 405
页数:3
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