Hematopoietic stem cell transplantation for chronic granulomatous disease.
Chronic granulomatous disease (CGD) is a primary immunodeficiency disease that is caused by the lack of 1 of 5 subunits of the superoxide-producing nicotinamide adenine dinucleotide phosphate oxidase of neutrophils, macrophages, and eosinophils. Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only curative treatment for CGD and can be offered to selected patients. Improved outcome with supportive care and high clinical variability in the disease course, however, make selection of eligible patients for HSCT difficult. This article addresses recent progress in HSCT for CGD, delineates present limitations, and points to future developments.
Seger
Reinhard A
RA
Division of Immunology/Hematology/BMT, University Children's Hospital, Steinwiesstrasse 75, Zürich 8032, Switzerland. Reinhard.Seger@kispi.uzh.ch
D000818:Animals; D006084:Graft Rejection / Q000209:etiology / Q000517:prevention & control; D006086:Graft vs Host Disease / Q000209:etiology / Q000517:prevention & control; D006105:Granulomatous Disease, Chronic / Q000235:genetics / Q000276:immunology* / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000639:trends*; D006648:Histocompatibility; D006801:Humans; D019255:NADPH Oxidases / Q000172:deficiency; D010587:Phagocytosis / Q000235:genetics / Q000276:immunology; D019172:Transplantation Conditioning
D019255:NADPH Oxidases