title: Failure of repeated cyclophosphamide pulse therapy in childhood cerebral X-linked adrenoleukodystrophy.
abstract: Childhood cerebral X-linked adrenoleukodystrophy (ALD) remains one of the most devastating neurological diseases of childhood. Cardinal features are inflammatory cerebral demyelination that coincides with an advancing zone of gadolinium enhancement on brain MRI. Therapeutic options, including the role of antiinflammatory therapy, are limited and poorly understood. We report the failure of repeated cyclophosphamide pulse therapy to halt the clinical progression or alter the gadolinium accumulation in two cases of childhood cerebral ALD.
authors.affiliation: Department of Biochemical Diseases, British Columbia Children's Hospital, Vancouver, British Columbia, Canada. ghorvath@cw.bc.ca
authors.forename: Gabriella Ana
authors.initials: GA
authors.lastname: Horvath
authors.forename: Florian
authors.initials: F
authors.lastname: Eichler
authors.forename: Ken
authors.initials: K
authors.lastname: Poskitt
authors.forename: Sylvia
authors.initials: S
authors.lastname: Stockler-Ipsiroglu
mesh_terms: D000326:Adrenoleukodystrophy / Q000175:diagnosis / Q000188:drug therapy*; D002648:Child; D003520:Cyclophosphamide / Q000627:therapeutic use*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008279:Magnetic Resonance Imaging; D008297:Male; D017211:Treatment Failure
chemical_list: D007166:Immunosuppressive Agents; D003520:Cyclophosphamide