Neuropediatrics 2012; 43(01): 048-052
DOI: 10.1055/s-0032-1307455
Short Communication
Thieme Medical Publishers 333 Seventh Avenue, New York, NY 10001, USA.

Failure of Repeated Cyclophosphamide Pulse Therapy in Childhood Cerebral X-Linked Adrenoleukodystrophy

Gabriella Ana Horvath
1  Department of Biochemical Diseases, British Columbia Children's Hospital, Vancouver, British Columbia, Canada
,
Florian Eichler
2  Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, United States
,
Ken Poskitt
3  Department of Radiology, British Columbia Children's Hospital, Vancouver, British Columbia, Canada
,
Sylvia Stockler-Ipsiroglu
4  Department of Pediatrics, British Columbia Children's Hospital, Vancouver, British Columbia, Canada
› Author Affiliations
Further Information

Publication History

28 September 2011

11 December 2011

Publication Date:
19 March 2012 (online)

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.