Advances in treatment for chronic granulomatous disease.
Chronic granulomatous disease (CGD) is a rare congenital disorder resulting from a failure of neutrophils to produce oxidases. Patients are therefore prone to recurrent infections from various organisms including fungi and atypical bacteria. The mortality in patients with the X-linked form of CGD, the most common type, ranges from 3% to 5% per year and although management of infections has improved with advances in antimicrobial therapies, better methods are needed to be able to cure these patients. Peripheral blood stem cell or bone marrow transplantation, while curative, is not widely used due to the episodic nature of the infections and the belief by many that conservative management is preferable to the risks of transplantation. Still, as will be discussed, improvements in the field are making allogenic transplantation more desirable and tilting the risk benefit ratio in favor of this modality. Additionally, gene therapy, which has been a long touted method to cure CGD, has within the last 5-10 years become more and more of a reality and may be realized by the end of this decade.
Kang
Elizabeth M
EM
National Institutes of Health, Building 10-Room 6-3752, 10 Center Drive, Bethesda, Maryland 20892, USA. Ekang@niaid.nih.gov
Malech
Harry L
HL
D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D016026:Bone Marrow Transplantation; D002986:Clinical Trials as Topic; D015316:Genetic Therapy; D006105:Granulomatous Disease, Chronic / Q000235:genetics / Q000276:immunology / Q000628:therapy*; D006801:Humans; D019255:NADPH Oxidases / Q000235:genetics; D009504:Neutrophils / Q000276:immunology; D033581:Stem Cell Transplantation
D000305:Adrenal Cortex Hormones; D019255:NADPH Oxidases