title: Bone marrow transplantation for chronic granulomatous disease: long-term follow-up and review of literature.
abstract: Chronic granulomatous disease (CGD) is a heterogeneous group of disorders with defective respiratory burst activity in phagocytes which results in recurrent pyogenic infections. We report an 8-year-old boy with X-linked CGD who received an HLA-identical BMT from his sister. The nitroblue tetrazolium test returned to normal 3 months post transplant. Neutrophil engraftment has been stable for 7 years post BMT. Our patient was the eighth case of CGD successfully treated by BMT. Conditioning regimens in these patients have consisted mainly of BU and CY. We suggest that BMT is a safe and effective method of cure for patients with CGD. BMT should be considered for patients with HLA-identical siblings.
authors.affiliation: Division of Haematology and Oncology, Department of Paediatrics, The Chinese University of Hong Kong, Prince of Wales Hospital, Hong Kong.
authors.forename: T
authors.initials: T
authors.lastname: Leung
authors.forename: K
authors.initials: K
authors.lastname: Chik
authors.forename: C
authors.initials: C
authors.lastname: Li
authors.forename: M
authors.initials: M
authors.lastname: Shing
authors.forename: P
authors.initials: P
authors.lastname: Yuen
mesh_terms: D016026:Bone Marrow Transplantation; D002066:Busulfan; D002648:Child; D004396:Coloring Agents; D003520:Cyclophosphamide; D005260:Female; D006105:Granulomatous Disease, Chronic / Q000235:genetics / Q000628:therapy*; D006801:Humans; D008297:Male; D009580:Nitroblue Tetrazolium; D010084:Oxidation-Reduction; D019172:Transplantation Conditioning; D016896:Treatment Outcome; D014960:X Chromosome
chemical_list: D004396:Coloring Agents; D009580:Nitroblue Tetrazolium; D003520:Cyclophosphamide; D002066:Busulfan